Trial Outcomes & Findings for A Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY) (NCT NCT03694275)
NCT ID: NCT03694275
Last Updated: 2022-05-26
Results Overview
Seizure frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period multiplied by 28. Percent Change from Baseline is defined as (frequency of seizures per 28 days during maintenance period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
Maintenance Period: Weeks 9 to 20
Results posted on
2022-05-26
Participant Flow
Participants took part in the study at 8 investigative sites in the United States from 10 September 2018 to 31 July 2020.
Participants with a diagnosis of 15q duplication syndrome (Dup15q) or CDKL5 deficiency disorder (CDD) were enrolled in 2 cohorts to receive treatment with TAK-935 for up to 20 weeks Treatment Period (8-week Dose Optimization Period and 12-week Maintenance Period).
Participant milestones
| Measure |
Soticlestat Dup15q
Soticlestat tablets twice daily (BID) orally or via gastrostomy tube (G-tube)/ percutaneous endoscopic gastrostomy (PEG) tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Overall Study
STARTED
|
8
|
12
|
|
Overall Study
COMPLETED
|
8
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
| Measure |
Soticlestat Dup15q
Soticlestat tablets twice daily (BID) orally or via gastrostomy tube (G-tube)/ percutaneous endoscopic gastrostomy (PEG) tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
|
Overall Study
Reason not Specified
|
0
|
1
|
Baseline Characteristics
A Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)
Baseline characteristics by cohort
| Measure |
Soticlestat Dup15q
n=8 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=12 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
15.4 years
STANDARD_DEVIATION 6.00 • n=5 Participants
|
7.6 years
STANDARD_DEVIATION 5.30 • n=7 Participants
|
10.7 years
STANDARD_DEVIATION 6.70 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
20 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
8 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
19 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
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
8 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Height
|
148.54 cm
STANDARD_DEVIATION 14.965 • n=5 Participants
|
124.59 cm
STANDARD_DEVIATION 26.882 • n=7 Participants
|
134.17 cm
STANDARD_DEVIATION 25.412 • n=5 Participants
|
|
Weight
|
43.40 kg
STANDARD_DEVIATION 13.678 • n=5 Participants
|
26.28 kg
STANDARD_DEVIATION 12.511 • n=7 Participants
|
33.13 kg
STANDARD_DEVIATION 15.283 • n=5 Participants
|
|
Body Mass Index (BMI)
|
19.21 kg/m^2
STANDARD_DEVIATION 4.149 • n=5 Participants
|
16.00 kg/m^2
STANDARD_DEVIATION 1.874 • n=7 Participants
|
17.28 kg/m^2
STANDARD_DEVIATION 3.314 • n=5 Participants
|
PRIMARY outcome
Timeframe: Maintenance Period: Weeks 9 to 20Population: Modified Intent-to-Treat (mITT) Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period. Overall number analyzed are the number of participants with data available for analyses.
Seizure frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period multiplied by 28. Percent Change from Baseline is defined as (frequency of seizures per 28 days during maintenance period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease.
Outcome measures
| Measure |
Soticlestat Dup15q
n=8 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=11 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Maintenance Period
|
11.7 percent change
Interval -90.0 to 39.0
|
-23.6 percent change
Interval -100.0 to 107.0
|
SECONDARY outcome
Timeframe: Treatment Period: Weeks 0 to 20Population: mITT Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period.
Seizure frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period multiplied by 28. Percent Change from Baseline is defined as (frequency of seizures per 28 days during the treatment period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease.
Outcome measures
| Measure |
Soticlestat Dup15q
n=8 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=12 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Treatment Period
|
13.4 percent change
Interval -89.0 to 30.0
|
-13.6 percent change
Interval -93.0 to 48.0
|
SECONDARY outcome
Timeframe: Maintenance Period: Weeks 9 to 20Population: mITT Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period. Overall number analyzed is the number of participants with data available for analyses.
Responders are defined as having over 50% motor seizure reduction compared to Baseline. Percent reduction from Baseline (%) is defined as \[(Maintenance Period motor Seizure Frequency - Baseline Period motor Seizure Frequency) divided by Baseline motor Seizure Frequency\] multiplied by 100. Data is reported as reduction of 25%, 50%, 75% and 100% or more in motor seizures from Baseline.
Outcome measures
| Measure |
Soticlestat Dup15q
n=8 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=11 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Percentage of Participants Considered as Treatment Responders During the Maintenance Period
<=0% Reduction
|
62.5 percentage of participants
|
27.3 percentage of participants
|
|
Percentage of Participants Considered as Treatment Responders During the Maintenance Period
>0% to <25% Reduction
|
12.5 percentage of participants
|
27.3 percentage of participants
|
|
Percentage of Participants Considered as Treatment Responders During the Maintenance Period
>=25% to <50% Reduction
|
12.5 percentage of participants
|
18.2 percentage of participants
|
|
Percentage of Participants Considered as Treatment Responders During the Maintenance Period
>=50% to <75% Reduction
|
0 percentage of participants
|
18.2 percentage of participants
|
|
Percentage of Participants Considered as Treatment Responders During the Maintenance Period
>=75% to 100% Reduction
|
12.5 percentage of participants
|
9.1 percentage of participants
|
SECONDARY outcome
Timeframe: Treatment Period: Weeks 0 to 20Population: mITT Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period. Overall number analyzed are all participants whose analyses were conducted using observed values and no imputation was done for missing data.
Seizure frequency is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period. Percent Change from Baseline is defined as (frequency of seizures during Treatment period - frequency of seizures at Baseline) divided by frequency of seizures at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease. The data is reported only for CDD participants.
Outcome measures
| Measure |
Soticlestat Dup15q
n=6 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Percent Change From Baseline in Frequency of Motor Seizures Longer Than 5 Minutes in Participants With CDD
|
-54.0 percent change
Interval -86.0 to 11.0
|
—
|
SECONDARY outcome
Timeframe: Maintenance Period: Weeks 9 to 20Population: mITT Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period. Overall number analyzed is the number of participants with data available for analyses.
Seizure-free days is defined as number of days with zero motor seizure during the period the Maintenance Period divided by number of days participant was in the Maintenance Period.
Outcome measures
| Measure |
Soticlestat Dup15q
n=8 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=11 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Proportion of Motor Seizure-free Days in Participants During the Maintenance Period
|
0.1 days/28 days
Interval 0.0 to 1.0
|
0.1 days/28 days
Interval 0.0 to 1.0
|
SECONDARY outcome
Timeframe: Baseline to Week 20Population: mITT Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period. Number analyzed is the number of participants with data available for analyses at the given timepoint.
The CGI-S focuses on clinician's observations of the participant's cognitive, functional, and behavioral performance since the beginning of the study. The CGI-S is rated on a 7-point scale, with the severity of illness scale using a range of responses where, 1= normal, not at all ill, 2= borderline mentally ill, 3= mildly ill, 4= moderately ill, 5= markedly ill, 6= severely ill and 7=amongst the most extremely ill participants. Negative change from Baseline indicates improvement.
Outcome measures
| Measure |
Soticlestat Dup15q
n=8 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=12 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Change From Baseline in Clinician's Global Impression of Severity (CGI-S) Responses of Investigator
Baseline
|
5.0 score on scale
Interval 4.0 to 6.0
|
5.0 score on scale
Interval 4.0 to 6.0
|
|
Change From Baseline in Clinician's Global Impression of Severity (CGI-S) Responses of Investigator
Change From Baseline at Week 20
|
0.0 score on scale
Interval -2.0 to 0.0
|
0.0 score on scale
Interval -1.0 to 1.0
|
SECONDARY outcome
Timeframe: Week 20Population: mITT Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period. Overall number analyzed is the number of participants with data available for analyses at given timepoint.
CGI-Change (CGI-C) treatment response ratings should take account of both therapeutic efficacy and treatment-related AEs. Each component of the CGI is rated separately; the instrument does not yield a global score. The CGI-C is rated on a 5-point scale, where, 0 = marked improvement and no side-effects, 1 = marked improvement and minimal side-effects, 2 = no change, 3 = minimal improvement and marked side-effects and 4 = unchanged or worse and side-effects outweigh the therapeutic effect. Lower scores indicated improvement.
Outcome measures
| Measure |
Soticlestat Dup15q
n=6 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=12 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression
Week 20, Score 0
|
33.3 percentage of participants
|
33.3 percentage of participants
|
|
Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression
Week 20, Score 1
|
0 percentage of participants
|
33.3 percentage of participants
|
|
Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression
Week 20, Score 2
|
50.0 percentage of participants
|
33.3 percentage of participants
|
|
Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression
Week 20, Score 3
|
16.7 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression
Week 20, Score 4
|
0 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Week 20Population: mITT Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period. Overall number analyzed is the number of participants with data available for analyses at the given timepoint.
CGI-Change (CGI-C) treatment response ratings should take account of both therapeutic efficacy and treatment-related AEs. Each component of the CGI is rated separately; the instrument does not yield a global score. The CGI-C is rated on a 7-point scale where, 1 = very much improved, 2 = much improved, 3 = slightly improved, 4= no change, 5= slightly worse, 6= much worse and 7= very much worse and marked side-effects. Lower scores indicated improvement.
Outcome measures
| Measure |
Soticlestat Dup15q
n=6 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=12 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family
Week 20, Score 1
|
0 percentage of participants
|
16.7 percentage of participants
|
|
Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family
Week 20, Score 2
|
16.7 percentage of participants
|
25.0 percentage of participants
|
|
Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family
Week 20, Score 3
|
33.3 percentage of participants
|
50.0 percentage of participants
|
|
Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family
Week 20, Score 4
|
50.0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family
Week 20, Score 5
|
0 percentage of participants
|
8.3 percentage of participants
|
|
Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family
Week 20, Score 6
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family
Week 20, Score 7
|
0 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline to Week 20Population: mITT Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses at the given timepoint.
Outcome measures
| Measure |
Soticlestat Dup15q
n=8 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=11 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Change From Baseline of Plasma 24S-hydroxycholesterol (24HC) Levels
Baseline
|
57.08 ng/mL
Standard Deviation 24.195
|
115.29 ng/mL
Standard Deviation 73.587
|
|
Change From Baseline of Plasma 24S-hydroxycholesterol (24HC) Levels
Change from Baseline at Week 20
|
-34.71 ng/mL
Standard Deviation 16.578
|
-75.64 ng/mL
Standard Deviation 29.284
|
SECONDARY outcome
Timeframe: Baseline to Week 20Population: mITT Analysis Set included all participants who have received at least 1 dose of study drug and have been assessed for at least 1 day in the Treatment Period.
Seizure Frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure during the period seizures were assessed multiplied by 28. Positive change from Baseline indicates seizure increase and negative change from Baseline indicates seizure decrease.
Outcome measures
| Measure |
Soticlestat Dup15q
n=8 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=12 Participants
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Change From Baseline in Seizure Frequency in Participants Treated With TAK-935 as an Adjunctive Therapy
Baseline
|
128.4 seizures per 28 days
Interval 35.0 to 224.0
|
77.8 seizures per 28 days
Interval 10.0 to 374.0
|
|
Change From Baseline in Seizure Frequency in Participants Treated With TAK-935 as an Adjunctive Therapy
Change From Baseline
|
13.2 seizures per 28 days
Interval -31.0 to 67.0
|
-3.4 seizures per 28 days
Interval -210.0 to 28.0
|
Adverse Events
Soticlestat Dup15q
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Soticlestat CDD
Serious events: 2 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Soticlestat Dup15q
n=8 participants at risk
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=12 participants at risk
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Immune system disorders
Anaphylactic reaction
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Infections and infestations
Respiratory syncytial virus bronchiolitis
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
Other adverse events
| Measure |
Soticlestat Dup15q
n=8 participants at risk
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with Dup15q weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
Soticlestat CDD
n=12 participants at risk
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
|
|---|---|---|
|
Nervous system disorders
Somnolence
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Blood and lymphatic system disorders
Eosinophilia
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
33.3%
4/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Gastrointestinal disorders
Frequent bowel movements
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
General disorders
Fatigue
|
25.0%
2/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
General disorders
Asthenia
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
General disorders
Peripheral swelling
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
General disorders
Pain
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Immune system disorders
Seasonal allergy
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Infections and infestations
Ear infection
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
16.7%
2/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Infections and infestations
Nasopharyngitis
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Infections and infestations
Respiratory tract infection
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Infections and infestations
Sinusitis
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Infections and infestations
Upper respiratory tract infection
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Injury, poisoning and procedural complications
Face injury
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Injury, poisoning and procedural complications
Fall
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Blood bicarbonate decreased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
16.7%
2/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Activated partial thromboplastin time prolonged
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Anticonvulsant drug level increased
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Blood cholesterol increased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Blood triglycerides increased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
C-reactive protein increased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Electrocardiogram QT prolonged
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Electrocardiogram ST-T change
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Haematocrit increased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
International normalised ratio increased
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Low density lipoprotein increased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Oxygen saturation decreased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
Platelet count increased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Investigations
White blood cell count increased
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Seizure
|
25.0%
2/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Lethargy
|
25.0%
2/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Partial seizures
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
16.7%
2/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Tonic convulsion
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
16.7%
2/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Atonic seizures
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Balance disorder
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Drooling
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Hypersomnia
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Hypotonia
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Nervous system disorders
Tremor
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Psychiatric disorders
Agitation
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Psychiatric disorders
Initial insomnia
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Psychiatric disorders
Irritability
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Psychiatric disorders
Sleep disorder
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Renal and urinary disorders
Neurogenic bladder
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Renal and urinary disorders
Urinary retentio
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Apnoea
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Hyperventilation
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
0.00%
0/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
8.3%
1/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Skin and subcutaneous tissue disorders
Rash
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
16.7%
2/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
12.5%
1/8 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
0.00%
0/12 • From the first dose to 30 days post-last dose of study treatment (Up to 24 weeks)
At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The first study related publication will be a multi-center publication submitted within 24 months after conclusion or termination of a study at all sites. After such multi site publication, all proposed site publications and presentations will be submitted to sponsor for review 60 days in advance of publication. Site will remove Sponsor confidential information unrelated to study results. Sponsor can delay a proposed publication for another 60 days to preserve intellectual property.
- Publication restrictions are in place
Restriction type: OTHER