Trial Outcomes & Findings for Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping (NCT NCT03218995)
NCT ID: NCT03218995
Last Updated: 2021-12-09
Results Overview
TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was any untoward medical occurrence in a participant, which does not necessarily have a causal relationship with the study drug. Abnormalities presented at Baseline were considered AEs if they reoccurred after resolution or worsen during the AE collection period. An SAE was defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, a congenital anomaly/birth defect, or an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
Baseline up to Week 100
Results posted on
2021-12-09
Participant Flow
Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) featuring a deletion mutation amenable to exon 51 skipping were enrolled into 2 cohorts based on their age.
Participant milestones
| Measure |
Eteplirsen
Eteplirsen was administered once every 7 days by intravenous (IV) infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 milligrams/kilogram (mg/kg) eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|
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Overall Study
STARTED
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15
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Overall Study
Received at Least 1 Dose of Study Drug
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15
|
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Overall Study
COMPLETED
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15
|
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Overall Study
NOT COMPLETED
|
0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
Baseline characteristics by cohort
| Measure |
Overall Study
n=15 Participants
Eteplirsen was administered once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|
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Age, Continuous
|
28.5 months
STANDARD_DEVIATION 12.94 • n=5 Participants
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Age, Customized
In utero
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0 Participants
n=5 Participants
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|
Age, Customized
Preterm newborn infants (gestational age < 37 wks)
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0 Participants
n=5 Participants
|
|
Age, Customized
Newborns (0-27 days)
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0 Participants
n=5 Participants
|
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Age, Customized
Infants and toddlers (28 days-23 months)
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6 Participants
n=5 Participants
|
|
Age, Customized
Children (2-11 years)
|
9 Participants
n=5 Participants
|
|
Age, Customized
Adolescents (12-17 years)
|
0 Participants
n=5 Participants
|
|
Age, Customized
Adults (18-64 years)
|
0 Participants
n=5 Participants
|
|
Age, Customized
From 65-84 years
|
0 Participants
n=5 Participants
|
|
Age, Customized
85 years and over
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0 Participants
n=5 Participants
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|
Sex: Female, Male
Female
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0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
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0 Participants
n=5 Participants
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|
Race (NIH/OMB)
White
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9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
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1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
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5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Week 100Population: The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was any untoward medical occurrence in a participant, which does not necessarily have a causal relationship with the study drug. Abnormalities presented at Baseline were considered AEs if they reoccurred after resolution or worsen during the AE collection period. An SAE was defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, a congenital anomaly/birth defect, or an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section.
Outcome measures
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 Participants
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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Cohort 2: Age 6 to <24 Months
n=6 Participants
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|---|
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Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug
TEAE
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9 Participants
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6 Participants
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|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug
SAE
|
0 Participants
|
1 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug
AE leading to discontinuation from study drug
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0 Participants
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0 Participants
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PRIMARY outcome
Timeframe: Baseline up to Week 100Population: The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
Clinical laboratory parameters that were evaluated included * Any Grade ≥2 (moderate) or serious event without an alternative etiology that the Investigator deemed was related to study drug * Two consecutive drug-related serum creatinine levels ≥2\*upper limit of normal (ULN) without an alternative etiology * Creatine kinase (CK) levels \>50,000 units/liter (U/L) * A confirmed, unexplained, increase in gamma glutamyl transferase (GGT) \>3\*ULN and either an increase in bilirubin \>2\*ULN or nascent prothrombin time \>2\*ULN concurrently, without an alternative etiology
Outcome measures
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 Participants
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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Cohort 2: Age 6 to <24 Months
n=6 Participants
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|---|
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Number of Participants With at Least 1 Potentially Clinically Significant Clinical Safety Laboratory Abnormality
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9 Participants
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6 Participants
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PRIMARY outcome
Timeframe: Baseline up to Week 100Population: The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
The vital sign parameters that were evaluated included blood pressure, heart rate, respiration, and temperature. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section.
Outcome measures
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 Participants
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
|
Cohort 2: Age 6 to <24 Months
n=6 Participants
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|---|
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Number of Participants With at Least 1 Markedly Abnormal Vital Sign
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9 Participants
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6 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Week 100Population: The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. Data were not collected for this Outcome Measure.
Data not collected during the study for this Outcome Measure. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Weeks 8, 12, 24, 36, 48, 60, 72, 84, 96Population: The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
The ECG was manually reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the centrally read ECG report were clinically significant. Clinical significance was defined as any variation in ECG findings that had medical relevance resulting in an alteration in medical care. The ECHO was reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the ECHO report were clinically significant. Clinical significance was defined as any variation in ECHO findings that had medical relevance resulting in an alteration in medical care. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section.
Outcome measures
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 Participants
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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Cohort 2: Age 6 to <24 Months
n=6 Participants
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|---|
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Number of Participants With at Least 1 Markedly Abnormal Electrocardiogram (ECG) and Echocardiogram (ECHO)
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4 Participants
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5 Participants
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SECONDARY outcome
Timeframe: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)Population: The Safety Set was used for pharmacokinetic(s) (PK) analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, 'Number Analyzed' signifies number of participants evaluable at the specified timepoint.
Outcome measures
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 Participants
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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Cohort 2: Age 6 to <24 Months
n=6 Participants
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|---|
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Maximum Plasma Concentration (Cmax) of Eteplirsen
Week 2 (2 mg/kg)
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9.67 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 75.9
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4.22 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 120
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Maximum Plasma Concentration (Cmax) of Eteplirsen
Week 6 (10 mg/kg)
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46.5 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 72.3
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17.2 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 192
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Maximum Plasma Concentration (Cmax) of Eteplirsen
Week 8 (20 mg/kg)
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63.3 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 123
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85.0 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 67.6
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Maximum Plasma Concentration (Cmax) of Eteplirsen
Week 10 (30 mg/kg)
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93.7 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 55.5
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63.8 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 124
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Maximum Plasma Concentration (Cmax) of Eteplirsen
Week 24 (30 mg/kg)
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78.2 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 92.2
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59.7 microgram/milliliter (μg/mL)
Geometric Coefficient of Variation 82.7
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SECONDARY outcome
Timeframe: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)Population: The Safety Set was used for PK analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, 'Number Analyzed' signifies number of participants evaluable at the specified timepoint.
Outcome measures
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 Participants
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
|
Cohort 2: Age 6 to <24 Months
n=6 Participants
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|---|
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Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen
2 mg/kg (Week 2)
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0.58 hours (hr)
Interval 0.17 to 2.67
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0.58 hours (hr)
Interval 0.42 to 0.67
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Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen
10 mg/kg (Week 6)
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0.58 hours (hr)
Interval 0.47 to 4.25
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0.72 hours (hr)
Interval 0.58 to 3.32
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Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen
20 mg/kg (Week 8)
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0.78 hours (hr)
Interval 0.5 to 2.75
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0.73 hours (hr)
Interval 0.53 to 1.17
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Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen
30 mg/kg (Week 10)
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0.58 hours (hr)
Interval 0.5 to 1.48
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0.92 hours (hr)
Interval 0.5 to 2.75
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Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen
30 mg/kg (Week 24)
|
0.63 hours (hr)
Interval 0.42 to 6.83
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0.72 hours (hr)
Interval 0.58 to 1.83
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SECONDARY outcome
Timeframe: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)Population: The Safety Set was used for PK analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, 'Number Analyzed' signifies number of participants evaluable at the specified timepoint.
Outcome measures
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 Participants
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
|
Cohort 2: Age 6 to <24 Months
n=6 Participants
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|---|
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Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma
2 mg/kg (Week 2)
|
13.8 μg*hr/mL
Geometric Coefficient of Variation 118
|
6.13 μg*hr/mL
Geometric Coefficient of Variation 73.1
|
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Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma
10 mg/kg (Week 6)
|
56.1 μg*hr/mL
Geometric Coefficient of Variation 57.2
|
27.8 μg*hr/mL
Geometric Coefficient of Variation 113
|
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Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma
20 mg/kg (Week 8)
|
92.1 μg*hr/mL
Geometric Coefficient of Variation 94.7
|
81.4 μg*hr/mL
Geometric Coefficient of Variation 89.6
|
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Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma
30 mg/kg (Week 10)
|
119 μg*hr/mL
Geometric Coefficient of Variation 30.8
|
85.0 μg*hr/mL
Geometric Coefficient of Variation 114
|
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Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma
30 mg/kg (Week 24)
|
100 μg*hr/mL
Geometric Coefficient of Variation 42.5
|
89.6 μg*hr/mL
Geometric Coefficient of Variation 43.8
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SECONDARY outcome
Timeframe: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)Population: The Safety Set was used for PK analysis and included all participants who were enrolled and received at least 1 dose of eteplirsen during the study. PK data were not collected for participants who received 4 mg of eteplirsen. Here, 'Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable at the specified timepoint.
Amount of unchanged drug excreted in urine from time 0 to 4 hours after completion of dosing is reported.
Outcome measures
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 Participants
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
|
Cohort 2: Age 6 to <24 Months
n=6 Participants
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
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|---|---|---|
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Amount of Drug Eliminated in Urine
2 mg/kg dose (Week 2)
|
7720 μg
Standard Deviation 9060
|
1430 μg
Standard Deviation 1390
|
|
Amount of Drug Eliminated in Urine
10 mg/kg (Week 6)
|
56000 μg
Standard Deviation 73300
|
28700 μg
Standard Deviation 24100
|
|
Amount of Drug Eliminated in Urine
20 mg/kg (Week 8)
|
102000 μg
Standard Deviation 108000
|
65600 μg
Standard Deviation 47900
|
|
Amount of Drug Eliminated in Urine
30 mg/kg (Week 10)
|
263000 μg
Standard Deviation 209000
|
94700 μg
Standard Deviation 68500
|
|
Amount of Drug Eliminated in Urine
30 mg/kg (Week 24)
|
239000 μg
Standard Deviation 140000
|
147000 μg
Standard Deviation 132000
|
Adverse Events
Cohort 1: Age 24 to 48 Months
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Cohort 2: Age 6 to <24 Months
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 participants at risk
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
|
Cohort 2: Age 6 to <24 Months
n=6 participants at risk
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
|
|---|---|---|
|
Infections and infestations
Bronchiolitis
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
Other adverse events
| Measure |
Cohort 1: Age 24 to 48 Months
n=9 participants at risk
Participants aged 24 to 48 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
|
Cohort 2: Age 6 to <24 Months
n=6 participants at risk
Participants aged 6 to \<24 months old were administered eteplirsen once every 7 days by IV infusion starting on Day 1 for up to 96 weeks. The starting dose was 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants continued to receive eteplirsen at 30 mg/kg for the duration of the study.
|
|---|---|---|
|
Infections and infestations
Nasopharyngitis
|
77.8%
7/9 • Number of events 27 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
83.3%
5/6 • Number of events 19 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Rhinitis
|
44.4%
4/9 • Number of events 4 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
66.7%
4/6 • Number of events 9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Ear infection
|
33.3%
3/9 • Number of events 6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
50.0%
3/6 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Gastroenteritis
|
33.3%
3/9 • Number of events 6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
50.0%
3/6 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Bronchitis
|
22.2%
2/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
33.3%
2/6 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Influenza
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
33.3%
2/6 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Conjunctivitis
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Pharyngitis
|
11.1%
1/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
33.3%
2/6 • Number of events 4 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Eye infection
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Gastrointestinal viral infection
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Hordeolum
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Lower respiratory tract infection
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Molluscum contagiosum
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Otitis media acute
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Roseola
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Varicella
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
General disorders
Pyrexia
|
77.8%
7/9 • Number of events 21 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
100.0%
6/6 • Number of events 21 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
General disorders
Catheter site eczema
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
General disorders
Catheter site swelling
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
General disorders
Discomfort
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
General disorders
Fatigue
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
General disorders
Hyperpyrexia
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
General disorders
Infusion site extravasation
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
General disorders
Localised oedema
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Vomiting
|
88.9%
8/9 • Number of events 15 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
66.7%
4/6 • Number of events 4 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Diarrhoea
|
55.6%
5/9 • Number of events 6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
50.0%
3/6 • Number of events 9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Constipation
|
22.2%
2/9 • Number of events 8 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Teething
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
33.3%
2/6 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Abdominal pain
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
22.2%
2/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Dental caries
|
22.2%
2/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Dental discomfort
|
11.1%
1/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Faeces discoloured
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Gastrointestinal disorders
Toothache
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Fall
|
55.6%
5/9 • Number of events 5 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
33.3%
2/6 • Number of events 5 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Head injury
|
55.6%
5/9 • Number of events 7 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
33.3%
2/6 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Contusion
|
22.2%
2/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Laceration
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Post-traumatic pain
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
11.1%
1/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Excoriation
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Joint injury
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Lip injury
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Penis injury
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Spinal fracture
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Tongue injury
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Tracheal obstruction
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Injury, poisoning and procedural complications
Vaccination complication
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
77.8%
7/9 • Number of events 25 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
83.3%
5/6 • Number of events 14 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
44.4%
4/9 • Number of events 7 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
50.0%
3/6 • Number of events 6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
22.2%
2/9 • Number of events 9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
22.2%
2/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Rash
|
22.2%
2/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 4 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
50.0%
3/6 • Number of events 8 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Rash generalised
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
22.2%
2/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Eczema nummular
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Miliaria
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Pruritus generalised
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Rash macular
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Ear and labyrinth disorders
Ear pain
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
33.3%
2/6 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Ear and labyrinth disorders
Cerumen impaction
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Ear and labyrinth disorders
External ear inflammation
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Ear and labyrinth disorders
Inner ear inflammation
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Ear and labyrinth disorders
Tympanic membrane hyperaemia
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Nervous system disorders
Lethargy
|
22.2%
2/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
33.3%
2/6 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Nervous system disorders
Headache
|
22.2%
2/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Nervous system disorders
Psychomotor hyperactivity
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Metabolism and nutrition disorders
Iron deficiency
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
33.3%
2/6 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Metabolism and nutrition disorders
Hyposideraemia
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Metabolism and nutrition disorders
Increased appetite
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Eye disorders
Chalazion
|
11.1%
1/9 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Eye disorders
Eye irritation
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Eye disorders
Eye pruritus
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Eye disorders
Visual acuity reduced
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Investigations
Body temperature
|
11.1%
1/9 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Investigations
Body temperature increased
|
11.1%
1/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 3 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Investigations
Amylase increased
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Investigations
Blood iron decreased
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Investigations
Haemophilus test positive
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Investigations
Streptococcus test positive
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Psychiatric disorders
Abnormal behaviour
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Psychiatric disorders
Anger
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Psychiatric disorders
Initial insomnia
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Psychiatric disorders
Irritability
|
11.1%
1/9 • Number of events 2 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Psychiatric disorders
Personality change
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
33.3%
3/9 • Number of events 8 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Vascular disorders
Flushing
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Vascular disorders
Haematoma
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Vascular disorders
Hypertension
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Blood and lymphatic system disorders
Autoimmune neutropenia
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Blood and lymphatic system disorders
Hypochromic anaemia
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Congenital, familial and genetic disorders
Haemoglobinopathy
|
11.1%
1/9 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
0.00%
0/6 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Immune system disorders
Allergy to chemicals
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Renal and urinary disorders
Dysuria
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
|
Reproductive system and breast disorders
Genital cyst
|
0.00%
0/9 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
16.7%
1/6 • Number of events 1 • Baseline up to Week 100
The Safety Set included all participants who were enrolled and received at least 1 dose of eteplirsen during the study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER