Trial Outcomes & Findings for Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C (NCT NCT02612129)
NCT ID: NCT02612129
Last Updated: 2024-11-29
Results Overview
NPC disease severity was assessed based on the 5-domain NPC Clinical Severity Scale (NPCCSS). The 5-domain NPCCSS focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
50 participants
Primary outcome timeframe
Baseline to Month 12
Results posted on
2024-11-29
Participant Flow
The study was conducted at 14 sites in Denmark, France, Germany, Italy, Poland, Spain, Switzerland, United Kingdom, and United States.
To confirm the selected dose of arimoclomol for participants less than 12 years of age, the 28 participants below 12 years old received a single arimoclomol dose for PK evaluation before randomization and the start of continuous treatment. A total of 50 participants were randomized in the blinded phase (continuous dose phase) and the study is ongoing in an open-label period.
Participant milestones
| Measure |
Arimoclomol Single PK Dose
Participants less than 12 years received a single oral dose of arimoclomol capsule, based on participant's body weight, on Day 1.
|
Arimoclomol (12-month Double-blind Phase)
Participants received arimoclomol capsules, orally based on participant's body weight, three times a day (TID) for 12 months.
|
Placebo (12-month Double-blind Phase)
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|---|
|
Single Dose Treatment Period
STARTED
|
28
|
0
|
0
|
|
Single Dose Treatment Period
COMPLETED
|
27
|
0
|
0
|
|
Single Dose Treatment Period
NOT COMPLETED
|
1
|
0
|
0
|
|
Continuous Treatment Period
STARTED
|
0
|
34
|
16
|
|
Continuous Treatment Period
COMPLETED
|
0
|
27
|
15
|
|
Continuous Treatment Period
NOT COMPLETED
|
0
|
7
|
1
|
Reasons for withdrawal
| Measure |
Arimoclomol Single PK Dose
Participants less than 12 years received a single oral dose of arimoclomol capsule, based on participant's body weight, on Day 1.
|
Arimoclomol (12-month Double-blind Phase)
Participants received arimoclomol capsules, orally based on participant's body weight, three times a day (TID) for 12 months.
|
Placebo (12-month Double-blind Phase)
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|---|
|
Single Dose Treatment Period
Screening Failure
|
1
|
0
|
0
|
|
Continuous Treatment Period
Early Escape
|
0
|
2
|
0
|
|
Continuous Treatment Period
Participant's Parents/legal guardian have Withdrawn Informed Consent
|
0
|
1
|
0
|
|
Continuous Treatment Period
Safety Reasons
|
0
|
3
|
0
|
|
Continuous Treatment Period
Death
|
0
|
1
|
0
|
|
Continuous Treatment Period
Participant Meets Criteria for IMP Administration to be Stopped and Subsequent Withdrawal
|
0
|
0
|
1
|
Baseline Characteristics
Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
Baseline characteristics by cohort
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Total
n=50 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
11.5 years
STANDARD_DEVIATION 5.4 • n=5 Participants
|
10.2 years
STANDARD_DEVIATION 4.1 • n=7 Participants
|
11.1 years
STANDARD_DEVIATION 5.0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
24 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
32 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
48 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
32 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
45 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age at Diagnosis of First Neurological Symptom
|
5.05 years
STANDARD_DEVIATION 3.43 • n=5 Participants
|
5.22 years
STANDARD_DEVIATION 3.87 • n=7 Participants
|
5.10 years
STANDARD_DEVIATION 3.54 • n=5 Participants
|
|
5-domain Niemann-Pick Disease Type C Clinical Severity Scale (NPCCSS) Score
|
12.1 score on a scale
STANDARD_DEVIATION 6.9 • n=5 Participants
|
9.4 score on a scale
STANDARD_DEVIATION 6.4 • n=7 Participants
|
11.2 score on a scale
STANDARD_DEVIATION 6.8 • n=5 Participants
|
|
17-domain NPCCSS Except Hearing Domains
|
21.2 score on a scale
STANDARD_DEVIATION 11.5 • n=5 Participants
|
17.2 score on a scale
STANDARD_DEVIATION 11.3 • n=7 Participants
|
19.9 score on a scale
STANDARD_DEVIATION 11.4 • n=5 Participants
|
|
Participants Currently Treated With Miglustat
Yes
|
26 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
39 Participants
n=5 Participants
|
|
Participants Currently Treated With Miglustat
No
|
8 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Month 12Population: Full Analysis Set (FAS) included those participants who were randomized and who had received at least one dose of randomized treatment medication. Overall Number analyzed is the number of participants evaluated at a specified timepoint.
NPC disease severity was assessed based on the 5-domain NPC Clinical Severity Scale (NPCCSS). The 5-domain NPCCSS focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=27 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=15 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Change From Baseline in the Niemann-Pick Disease Type C (NPC) Disease Severity Assessed Based on the 5-domain NPCCSS Total Scores
|
0.76 score on a scale
Interval -0.05 to 1.56
|
2.15 score on a scale
Interval 1.05 to 3.25
|
SECONDARY outcome
Timeframe: Month 12Population: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication.
The CGI-I is a 7-point scale that rates total improvement of participant's condition. The clinician rates the participants from 1=Very much improved, 2=Much improved, 3=Minimally improved, 4=No change, 5=Minimally worse, 6=Much worse, or 7=Very much worse. Scores thus range from 1-7 with lower scores indicating greater improvement. Responders were the participants with a score of 1 or 2 at Month 12.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Percentage of Responders in Clinical Global Impression Scale of Improvement (CGI-I) - Defined as Percentage of Participants Where the CGI-I Score Remains Stable or Shows Improvement (This Outcome Measure Was Considered Co-primary by the FDA)
|
58.8 percentage of responders
|
56.3 percentage of responders
|
SECONDARY outcome
Timeframe: Baseline to Month 12Population: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication.
NPC disease severity was assessed based on the 5-domain NPC Clinical Severity Scale (NPCCSS). The 5-domain NPCCSS focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment. Stable was defined as a participant's total score for the 5 domains being the same at month 12 as at baseline. Improvement was defined as a participant's total score at month 12 being lower than at baseline.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Percentage of Responders in 5-domain NPCCSS - Defined as Participants Where the 5-domain NPCCSS Score Remains Stable or Improves as Compared to Baseline
|
50.0 percentage of responders
|
37.5 percentage of responders
|
SECONDARY outcome
Timeframe: Baseline to Month 12Population: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication.
Time to worsening was defined as the time until the participant reached the predefined minimal clinically important difference (MCID) of 2 points compared to baseline on the 5-domain NPC Clinical Severity Scale (NPCCSS). The 5-domain NPCCSS focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment. The values reported per group are the 25th percentile Kaplan-Meier estimates and 95% confidence interval.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Time to Worsening
|
5.2 months
Interval 2.9 to 12.0
|
5.5 months
Interval 1.0 to 6.5
|
SECONDARY outcome
Timeframe: Months 6 and 12Population: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication.
Worsening was defined as participants that have reached the predefined MCID of 2 points on their 5-domain NPC Clinical Severity Scale (NPCCSS). The 5-domain NPCCSS focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Percentage of Participants With Worsening
Worsening at Month 6
|
35.3 percentage of participants
|
50.0 percentage of participants
|
|
Percentage of Participants With Worsening
Worsening at Month 12
|
44.1 percentage of participants
|
43.8 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline to 6 and 12 monthsPopulation: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. Here, "Number analyzed" signifies number of participants evaluated at specified timepoint.
The NPC Clinical Severity Scale (NPCCSS) is a disease-specific, clinician-reported outcome measure developed to characterize and quantify NPC disease progression. The 17-domain NPCCSS includes clinical signs and symptoms in nine major and eight minor domains, which are rated on scales of 0-5 (for the major domains) or 0-2 (for the minor domains). The total score is the sum of the score of each of the 17 domains and ranges from 0 to 61, with a high score indicating a more severe clinical impairment.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Change From Baseline in 17-domain NPCCSS Apart From Hearing Domains (i.e. Hearing and Auditory Brainstem Response)
Change at Month 6
|
0.53 score on a scale
Interval -0.85 to 1.9
|
2.22 score on a scale
Interval 0.33 to 4.1
|
|
Change From Baseline in 17-domain NPCCSS Apart From Hearing Domains (i.e. Hearing and Auditory Brainstem Response)
Change at Month 12
|
1.20 score on a scale
Interval -0.4 to 2.79
|
2.81 score on a scale
Interval 0.75 to 4.87
|
SECONDARY outcome
Timeframe: Baseline to 6 monthsPopulation: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication.
The 5-domain NPC Clinical Severity Scale (NPCCSS) focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Change From Baseline in 5-domain NPCCSS Score
|
0.48 score on a scale
Interval -0.05 to 1.02
|
1.60 score on a scale
Interval 0.86 to 2.34
|
SECONDARY outcome
Timeframe: Baseline to 6 and 12 monthsPopulation: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. Here, "Number analyzed" signifies number of participants evaluated at specified timepoint.
The NPC Clinical Severity Scale (NPCCSS) is a disease-specific, clinician-reported outcome measure developed to characterize and quantify disease progression. The 17-domain NPCCSS includes clinical signs and symptoms in nine major (ambulation, cognition, eye movement, fine motor, hearing, memory, seizures, speech, swallowing,) and eight minor (auditory brainstem response, behavior, gelastic cataplexy, hyperreflexia, incontinence, narcolepsy, psychiatric, respiratory problems) domains, which are rated on scales of 0-5 (for the major domains) or 0-2 (for the minor domains). A higher score indicates a more severe clinical impairment.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Ambulation: Baseline
|
2.5 score on a scale
Standard Deviation 1.6
|
2.2 score on a scale
Standard Deviation 1.6
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Ambulation: Change at Month 6
|
0.1 score on a scale
Standard Deviation 0.4
|
0.3 score on a scale
Standard Deviation 1.0
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Ambulation: Change at Month 12
|
0.3 score on a scale
Standard Deviation 0.5
|
0.3 score on a scale
Standard Deviation 0.9
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Speech: Baseline
|
2.2 score on a scale
Standard Deviation 1.6
|
1.6 score on a scale
Standard Deviation 1.2
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Speech: Change at Month 6
|
0.1 score on a scale
Standard Deviation 0.5
|
0.1 score on a scale
Standard Deviation 0.3
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Speech: Change at Month 12
|
-0.2 score on a scale
Standard Deviation 1.0
|
0.3 score on a scale
Standard Deviation 0.8
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Swallow: Baseline
|
1.9 score on a scale
Standard Deviation 1.7
|
1.3 score on a scale
Standard Deviation 1.7
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Swallow: Change at Month 6
|
0.1 score on a scale
Standard Deviation 1.1
|
0.4 score on a scale
Standard Deviation 1.0
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Swallow: Change at Month 12
|
0.1 score on a scale
Standard Deviation 1.1
|
0.6 score on a scale
Standard Deviation 1.0
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Fine Motor Skills: Baseline
|
2.8 score on a scale
Standard Deviation 1.8
|
1.9 score on a scale
Standard Deviation 1.8
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Fine Motor Skills: Change at Month 6
|
0.1 score on a scale
Standard Deviation 0.7
|
0.5 score on a scale
Standard Deviation 1.1
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Fine Motor Skills: Change at Month 12
|
0.2 score on a scale
Standard Deviation 0.8
|
0.6 score on a scale
Standard Deviation 1.3
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Cognition: Baseline
|
2.8 score on a scale
Standard Deviation 1.3
|
2.5 score on a scale
Standard Deviation 1.5
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Cognition: Change at Month 6
|
0.2 score on a scale
Standard Deviation 0.5
|
0.3 score on a scale
Standard Deviation 0.8
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Cognition: Change at Month 12
|
0.3 score on a scale
Standard Deviation 0.6
|
0.1 score on a scale
Standard Deviation 0.6
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Eye Movement: Baseline
|
2.2 score on a scale
Standard Deviation 1.2
|
2.1 score on a scale
Standard Deviation 1.1
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Eye Movement: Change at Month 6
|
0.0 score on a scale
Standard Deviation 0.6
|
-0.1 score on a scale
Standard Deviation 0.7
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Eye Movement: Change at Month 12
|
0.2 score on a scale
Standard Deviation 0.9
|
-0.1 score on a scale
Standard Deviation 0.6
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Memory: Baseline
|
1.9 score on a scale
Standard Deviation 1.4
|
1.3 score on a scale
Standard Deviation 1.5
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Memory: Change at Month 6
|
0.1 score on a scale
Standard Deviation 0.5
|
0.2 score on a scale
Standard Deviation 1.1
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Memory: Change at Month 12
|
0.1 score on a scale
Standard Deviation 0.5
|
0.3 score on a scale
Standard Deviation 0.8
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Seizures: Baseline
|
1.9 score on a scale
Standard Deviation 1.9
|
1.3 score on a scale
Standard Deviation 1.8
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Seizures: Change at Month 6
|
-0.1 score on a scale
Standard Deviation 0.8
|
0.0 score on a scale
Standard Deviation 1.5
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Seizures: Change at Month 12
|
0.3 score on a scale
Standard Deviation 0.8
|
-0.1 score on a scale
Standard Deviation 1.7
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Hearing: Baseline
|
0.4 score on a scale
Standard Deviation 1.0
|
0.0 score on a scale
Standard Deviation 0.0
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Hearing: Change at Month 6
|
-0.2 score on a scale
Standard Deviation 0.8
|
0.0 score on a scale
Standard Deviation 0.0
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Hearing: Change at Month 12
|
0.0 score on a scale
Standard Deviation 0.0
|
0.3 score on a scale
Standard Deviation 0.8
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Auditory Brainstem Response: Baseline
|
0.2 score on a scale
Standard Deviation 0.4
|
0.1 score on a scale
Standard Deviation 0.4
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Auditory Brainstem Response: Change at Month 6
|
0.0 score on a scale
Standard Deviation 0.0
|
0.2 score on a scale
Standard Deviation 0.4
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Auditory Brainstem Response: Change at Month 12
|
0.0 score on a scale
Standard Deviation 0.0
|
0.0 score on a scale
Standard Deviation 0.0
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Behavior: Baseline
|
0.4 score on a scale
Standard Deviation 0.6
|
0.4 score on a scale
Standard Deviation 0.6
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Behavior: Change at Month 6
|
0.0 score on a scale
Standard Deviation 0.5
|
-0.1 score on a scale
Standard Deviation 0.5
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Behavior: Change at Month 12
|
0.1 score on a scale
Standard Deviation 0.6
|
-0.2 score on a scale
Standard Deviation 0.4
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Gelastic Cataplexy: Baseline
|
0.8 score on a scale
Standard Deviation 0.9
|
0.4 score on a scale
Standard Deviation 0.8
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Gelastic Cataplexy: Change at Month 6
|
0.1 score on a scale
Standard Deviation 0.5
|
0.3 score on a scale
Standard Deviation 0.6
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Gelastic Cataplexy: Change at Month 12
|
0.2 score on a scale
Standard Deviation 0.6
|
0.3 score on a scale
Standard Deviation 0.6
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Hyperreflexia: Baseline
|
1.0 score on a scale
Standard Deviation 0.7
|
1.1 score on a scale
Standard Deviation 0.9
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Hyperreflexia: Change at Month 6
|
-0.0 score on a scale
Standard Deviation 0.5
|
0.2 score on a scale
Standard Deviation 0.6
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Hyperreflexia: Change at Month 12
|
0.1 score on a scale
Standard Deviation 0.7
|
0.1 score on a scale
Standard Deviation 0.5
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Incontinence: Baseline
|
1.0 score on a scale
Standard Deviation 0.8
|
0.8 score on a scale
Standard Deviation 0.9
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Incontinence: Change at Month 6
|
-0.1 score on a scale
Standard Deviation 0.4
|
0.1 score on a scale
Standard Deviation 0.5
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Incontinence: Change at Month 12
|
-0.0 score on a scale
Standard Deviation 0.4
|
0.1 score on a scale
Standard Deviation 0.9
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Narcolepsy (NARCO): Baseline
|
0.1 score on a scale
Standard Deviation 0.4
|
0.3 score on a scale
Standard Deviation 0.7
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Narcolepsy (NARCO): Change at Month 6
|
0.0 score on a scale
Standard Deviation 0.4
|
-0.1 score on a scale
Standard Deviation 0.3
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Narcolepsy (NARCO): Change at Month 12
|
-0.1 score on a scale
Standard Deviation 0.4
|
-0.1 score on a scale
Standard Deviation 0.3
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Psychiatric: Baseline
|
0.1 score on a scale
Standard Deviation 0.4
|
0.1 score on a scale
Standard Deviation 0.5
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Psychiatric: Change at Month 6
|
-0.0 score on a scale
Standard Deviation 0.3
|
0.1 score on a scale
Standard Deviation 0.6
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Psychiatric: Change at Month 12
|
0.0 score on a scale
Standard Deviation 0.3
|
0.0 score on a scale
Standard Deviation 0.4
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Respiratory: Baseline
|
0.1 score on a scale
Standard Deviation 0.3
|
0.0 score on a scale
Standard Deviation 0.0
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Respiratory: Change at Month 6
|
0.0 score on a scale
Standard Deviation 0.5
|
0.1 score on a scale
Standard Deviation 0.3
|
|
Changes From Baseline in Each Individual Domain of the NPCCSS
Respiratory: Change at Month 12
|
0.1 score on a scale
Standard Deviation 0.5
|
0.2 score on a scale
Standard Deviation 0.4
|
SECONDARY outcome
Timeframe: Baseline to 6 and 12 monthsPopulation: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication.
The NPC Clinical Database (NPC-cdb) score aims to reflect the current clinical status of the participant. The NPC-cdb score represents both historical symptoms and a current status. The test consists of ten areas: visceral signs, development, motor function, ocular-motor abnormalities, seizures/cataplexy/narcolepsy, cognitive abilities and memory, behavioral and psychiatric abnormalities, speech, hearing, and abilities in daily life. The current status score is a severity-weighted sum of 72 symptoms considered as disease-relevant at the time of assessment. Each symptom contributes with a score between 1 and 5, the maximum score is 125. An increase in score reflects a reduction in the participant's abilities.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Change From Baseline in the NPC Clinical Database (NPC-CDB) Score (Modified "Stampfer Score")
Change at Month 6
|
-0.38 score on a scale
Interval -3.3 to 2.54
|
4.71 score on a scale
Interval 0.49 to 8.93
|
|
Change From Baseline in the NPC Clinical Database (NPC-CDB) Score (Modified "Stampfer Score")
Change at Month 12
|
1.85 score on a scale
Interval -2.16 to 5.86
|
4.88 score on a scale
Interval -0.63 to 10.39
|
SECONDARY outcome
Timeframe: Baseline to 6 and 12 monthsPopulation: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. Here, "Overall Number of Participants Analyzed" is the number of participants with data for the outcome measure and "Number analyzed" signifies number of participants evaluated at specified timepoint.
The EQ-5D-Y descriptive system includes 5 descriptive items: Mobility, self-care, doing usual activities, having pain or discomfort, and feeling anxiety or depressed. Each dimension has 3 levels: No problems, some problems, and a lot of problems. The change in the 5 individual items of the EQ-5D-Y per participant was explored by using the pareto principle at 6 and 12 months to show the number (%) of participants who felt: * Better (better on at least one dimension and no worse in any other dimension), * Worse (worse in at least one dimension, and no better in any other dimension)
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=30 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=15 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Percentage of Participants With Change From Baseline in Quality of Life (EQ-5D-Y)
Better: Change at Month 6
|
16.7 percentage of participants
|
26.7 percentage of participants
|
|
Percentage of Participants With Change From Baseline in Quality of Life (EQ-5D-Y)
Worse: Change at Month 6
|
40.0 percentage of participants
|
46.7 percentage of participants
|
|
Percentage of Participants With Change From Baseline in Quality of Life (EQ-5D-Y)
Better: Change at Month 12
|
25.9 percentage of participants
|
40.0 percentage of participants
|
|
Percentage of Participants With Change From Baseline in Quality of Life (EQ-5D-Y)
Worse: Change at Month 12
|
44.4 percentage of participants
|
20.0 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline to 6 and 12 monthsPopulation: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication.
The SARA included eight items reflecting neurologic manifestations of cerebellar ataxia. The test provides a direct and simple description of motor function in a participant. The test consists of 8 test items: gait, stance, sitting, speech disturbance, finger chase, nose-finger test, fast alternating hand movements, and heel-shin slide. The total score of the 8 items ranges from 0 (normal cerebellar function) to 40 (not able to perform any of the test items).
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Change From Baseline in the Scale for Assessment and Rating of Ataxia (SARA) Score
Change at 6 months
|
0.79 score on a scale
Interval -0.16 to 1.75
|
0.05 score on a scale
Interval -1.29 to 1.4
|
|
Change From Baseline in the Scale for Assessment and Rating of Ataxia (SARA) Score
Change at 12 months
|
1.06 score on a scale
Interval -0.17 to 2.29
|
0.78 score on a scale
Interval -0.9 to 2.47
|
SECONDARY outcome
Timeframe: Baseline to 6 and 12 monthsPopulation: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication.
The 9HPT test is a direct and simple measurement of fine motor coordination function, eye/hand coordination, and the ability to follow a simple direction. The 9HPT is a timed test in which nine pegs are inserted and removed from nine holes in the pegboard. Both hands are tested starting with the dominant hand. The time spent in completing the 9 HPT using each hand was recorded.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=34 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Change From Baseline in the Time Spent to Complete the Nine-Hole Peg Test (9HPT)
Dominant Hand: Change at 6 months
|
1.54 seconds
Interval -24.98 to 28.06
|
11.88 seconds
Interval -20.88 to 44.64
|
|
Change From Baseline in the Time Spent to Complete the Nine-Hole Peg Test (9HPT)
Non-dominant Hand: Change at 6 months
|
0.60 seconds
Interval -27.25 to 28.45
|
16.46 seconds
Interval -17.07 to 50.0
|
|
Change From Baseline in the Time Spent to Complete the Nine-Hole Peg Test (9HPT)
Dominant Hand: Change at 12 months
|
-3.29 seconds
Interval -15.56 to 8.98
|
-6.49 seconds
Interval -20.34 to 7.37
|
|
Change From Baseline in the Time Spent to Complete the Nine-Hole Peg Test (9HPT)
Non-dominant Hand: Change at 12 months
|
11.68 seconds
Interval -14.89 to 38.25
|
17.59 seconds
Interval -13.24 to 48.42
|
SECONDARY outcome
Timeframe: Months 6 and 12Population: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. Here, "Overall Number of Participants Analyzed" is the number of participants with data for the outcome measure and "Number analyzed" signifies number of participants evaluated at specified timepoint.
The CGI-S is a 7-point scale that requires the clinician to rate the severity of the participant's illness at the time of assessment. A rating of 1 is considered normal, or with the least severe symptoms, a rating of 7 is extremely ill, or the worst symptoms. Scores thus range from 1-7 with lower scores indicating less severe disease.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=29 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=15 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 6: Normal, not ill at all
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 6: Borderline ill
|
10.3 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 6: Mildly ill
|
27.6 percentage of participants
|
28.6 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 6: Moderately ill
|
10.3 percentage of participants
|
35.7 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 6: Markedly ill
|
31.0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 6: Severely ill
|
20.7 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 6: Most extremely ill participants
|
0 percentage of participants
|
7.1 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 12: Normal, not ill at all
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 12: Borderline ill
|
7.4 percentage of participants
|
6.7 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 12: Mildly ill
|
29.6 percentage of participants
|
26.7 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 12: Moderately ill
|
11.1 percentage of participants
|
33.3 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 12: Markedly ill
|
29.6 percentage of participants
|
6.7 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 12: Severely ill
|
22.2 percentage of participants
|
26.7 percentage of participants
|
|
Percentage of Participants Within Each Severity Category of the Clinical Global Impression Scale of Severity (CGI-S)
Month 12: Most extremely ill participants
|
0 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Months 6 and 12Population: FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. Here, "Overall Number of Participants Analyzed" is the number of participants with data for the outcome measure and "Number analyzed" signifies number of participants evaluated at specified timepoint.
The CGI-I is a 7-point scale that rates total improvement of participant's condition. The clinician rates the participants from 1=Very much improved, 2=Much improved, 3=Minimally improved, 4=No change, 5=Minimally worse, 6=Much worse, or 7=Very much worse. Scores thus range from 1-7 with lower scores indicating greater improvement.
Outcome measures
| Measure |
Arimoclomol (12-month Double-blind Phase)
n=29 Participants
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=15 Participants
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 12: Very Much Worse
|
3.7 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 6: Very Much Improved
|
3.4 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 6: Much Improved
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 6: Minimally Improved
|
24.1 percentage of participants
|
25.0 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 6: No Change
|
37.9 percentage of participants
|
25.0 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 6: Minimally Worse
|
27.6 percentage of participants
|
33.3 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 6: Much Worse
|
6.9 percentage of participants
|
16.7 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 6: Very Much Worse
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 12: Very Much Improved
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 12: Much Improved
|
3.7 percentage of participants
|
6.7 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 12: Minimally Improved
|
18.5 percentage of participants
|
33.3 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 12: No Change
|
51.9 percentage of participants
|
20.0 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 12: Minimally Worse
|
14.8 percentage of participants
|
13.3 percentage of participants
|
|
Percentage of Participants Within Each Category of the Clinical Global Impression Scale of Improvement (CGI-I)
Month 12: Much Worse
|
7.4 percentage of participants
|
26.7 percentage of participants
|
Adverse Events
Arimoclomol Single PK Dose
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Arimoclomol (12-month Double-blind Phase)
Serious events: 5 serious events
Other events: 30 other events
Deaths: 1 deaths
Placebo (12-month Double-blind Phase)
Serious events: 6 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Arimoclomol Single PK Dose
n=28 participants at risk
Participants less than 12 years received a single oral dose of arimoclomol capsule, based on participant's body weight, on Day 1.
|
Arimoclomol (12-month Double-blind Phase)
n=34 participants at risk
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 participants at risk
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|---|
|
Cardiac disorders
Cardio-respiratory Arrest
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Lower Respiratory Tract Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Pneumonia
|
3.6%
1/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
12.5%
2/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Craniocerebral Injury
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Laceration
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Metabolism and nutrition disorders
Hypophagia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Metabolism and nutrition disorders
Malnutrition
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Musculoskeletal and connective tissue disorders
Foot Deformity
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Epilepsy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
12.5%
2/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Epileptic Encephalopathy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Distress
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Skin and subcutaneous tissue disorders
Angioedema
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Investigations
Aspiration Bronchial
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Seizure
|
3.6%
1/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Respiratory Tract Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
Other adverse events
| Measure |
Arimoclomol Single PK Dose
n=28 participants at risk
Participants less than 12 years received a single oral dose of arimoclomol capsule, based on participant's body weight, on Day 1.
|
Arimoclomol (12-month Double-blind Phase)
n=34 participants at risk
Participants received arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
Placebo (12-month Double-blind Phase)
n=16 participants at risk
Participants received matching placebo to arimoclomol capsules, orally based on participant's body weight, TID for 12 months.
|
|---|---|---|---|
|
Psychiatric disorders
Agitation
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Psychiatric disorders
Hallucination, auditory
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
3.6%
1/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
General disorders
Pyrexia
|
3.6%
1/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
17.6%
6/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
18.8%
3/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Immune system disorders
Drug Hypersensitivity
|
3.6%
1/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
3.6%
1/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
17.6%
6/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Skin and subcutaneous tissue disorders
Rash
|
3.6%
1/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Congenital, familial and genetic disorders
Phimosis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Ear and labyrinth disorders
Deafness Neurosensory
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Ear and labyrinth disorders
Deafness Unilateral
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Ear and labyrinth disorders
Ear Pain
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Endocrine disorders
Hypothyroidism
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Eye disorders
Pupils Unequal
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Abdominal Discomfort
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Abdominal Distension
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Aphthous Ulcer
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
20.6%
7/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
18.8%
3/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Dental Caries
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
20.6%
7/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
12.5%
2/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Gastrooesophageal Reflux Disease
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Lip Dry
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Mouth Cyst
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Odynophagia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Salivary Hypersecretion
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
23.5%
8/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
25.0%
4/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
General disorders
Asthenia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
General disorders
Fatigue
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
General disorders
Gait Disturbance
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
General disorders
Medical Device Site Dermatitis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
General disorders
Pain
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
General disorders
Peripheral Swelling
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
General disorders
Secretion Discharge
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Hepatobiliary disorders
Hepatic Lesion
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Immune system disorders
Food Allergy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Immune system disorders
Seasonal Allergy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Bronchitis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
11.8%
4/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
12.5%
2/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Conjunctivitis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Ear Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
12.5%
2/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Eye Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
12.5%
2/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Fungal Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
12.5%
2/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Gastroenteritis Viral
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Hand-foot-and-mouth Disease
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Hordeolum
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Influenza
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Localised Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Lower Respiratory Tract Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
8.8%
3/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Medical Device Site Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
25.0%
4/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Onychomycosis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Oral Candidiasis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Oral Fungal Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Respiratory Tract Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
8.8%
3/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Rhinitis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
14.7%
5/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
12.5%
2/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Skin Candida
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Viral Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Viral Upper Respiratory Tract Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Vulvovaginal Mycotic Infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Infections and infestations
Wound infection
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Eschar
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Head Injury
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Injury
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Joint Dislocation
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Laceration
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Ligament Sprain
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Procedural Pain
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Injury, poisoning and procedural complications
Tooth Injury
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Investigations
Blood Creatinine Increased
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Investigations
Body Temperature Abnormal
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Investigations
Body Temperature Increased
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Investigations
C-reactive Protein Increased
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Investigations
Vitamin D Decreased
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Investigations
Weight Decreased
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
14.7%
5/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
8.8%
3/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Metabolism and nutrition disorders
Iron Deficiency
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Metabolism and nutrition disorders
Refeeding Syndrome
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Metabolism and nutrition disorders
Vitamin C Deficiency
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Musculoskeletal and connective tissue disorders
Coccydynia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Aphasia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Cognitive Disorder
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Epilepsy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
12.5%
2/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Headache
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
8.8%
3/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Lethargy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Memory Impairment
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Migraine
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Motor Dysfunction
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Muscle Spasticity
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Peroneal Nerve Palsy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Petit Mal Epilepsy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Quadriplegia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Seizure
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
8.8%
3/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Speech Disorder
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Tremor
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Psychiatric disorders
Aggression
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Psychiatric disorders
Hallucination, visual
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Psychiatric disorders
Sleep Disorder
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Renal and urinary disorders
Oliguria
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Reproductive system and breast disorders
Premenstrual Syndrome
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial Lung Disease
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
5.9%
2/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
8.8%
3/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Productive Cough
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Sleep Apnoea Syndrome
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Skin and subcutaneous tissue disorders
Acne
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Skin and subcutaneous tissue disorders
Angioedema
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Skin and subcutaneous tissue disorders
Dandruff
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Skin and subcutaneous tissue disorders
Henoch-Schonlein Purpura
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Surgical and medical procedures
Gastrostomy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Vascular disorders
Haematoma
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
2.9%
1/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Nervous system disorders
Cataplexy
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
|
Psychiatric disorders
Depression
|
0.00%
0/28 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
0.00%
0/34 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
6.2%
1/16 • From first dose of study drug up to last dose of blinded study treatment (Month 12)
Safety analysis set included all participants who had received at least one dose of study drug. Adverse events were reported separately for the single dose period where 28 participants less than 12 years old received a single dose of arimoclomol, and the 12-month continuous dose period where all participants received randomized treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER