Trial Outcomes & Findings for A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (NCT NCT04336826)
NCT ID: NCT04336826
Last Updated: 2024-04-01
Results Overview
An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. Serious adverse event (SAE): an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, important medical event. A TEAE was defined as an AE that occurred or worsened while on ataluren (on or after first dose of ataluren) up to 4 weeks after the last dose. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
COMPLETED
PHASE2
6 participants
Baseline up to Week 28
2024-04-01
Participant Flow
Participant milestones
| Measure |
Ataluren
Participants received ataluren oral suspension 10 milligrams (mg)/kilogram (kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for up to 24 weeks.
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|---|---|
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Overall Study
STARTED
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6
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Overall Study
Received at Least 1 Dose of Study Drug
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6
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Overall Study
COMPLETED
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6
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Overall Study
NOT COMPLETED
|
0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Baseline characteristics by cohort
| Measure |
Ataluren
n=6 Participants
Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for up to 24 weeks.
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|---|---|
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Age, Continuous
|
1.153 years
STANDARD_DEVIATION 0.4485 • n=5 Participants
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Sex: Female, Male
Female
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0 Participants
n=5 Participants
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Sex: Female, Male
Male
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6 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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5 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
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Race (NIH/OMB)
Black or African American
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0 Participants
n=5 Participants
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Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
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Race (NIH/OMB)
More than one race
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1 Participants
n=5 Participants
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Race (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Baseline up to Week 28Population: Safety analysis set included all participants who received at least 1 dose of ataluren.
An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. Serious adverse event (SAE): an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, important medical event. A TEAE was defined as an AE that occurred or worsened while on ataluren (on or after first dose of ataluren) up to 4 weeks after the last dose. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
Ataluren
n=6 Participants
Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for up to 24 weeks.
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|---|---|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
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5 Participants
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SECONDARY outcome
Timeframe: Predose up to 12 hours postdose at Week 24Population: PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
Ataluren
n=4 Participants
Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for up to 24 weeks.
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|---|---|
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Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren
|
167 hours*micrograms (μg)/milliliter (mL)
Standard Deviation 23.8
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SECONDARY outcome
Timeframe: Predose up to 12 hours postdose at Week 24Population: PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum.
Outcome measures
| Measure |
Ataluren
n=6 Participants
Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for up to 24 weeks.
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|---|---|
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Area Under the Concentration-Time Curve Between Dosing Interval (AUC0-τ) of Ataluren
|
NA hours*μg/mL
Standard Deviation NA
Per prespecified analysis, 'Measure Type' and 'Method of Dispersion' were not estimable for this outcome measure because there were insufficient number of participants with data within the linear regression of concentration in the logarithmic scale versus time.
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SECONDARY outcome
Timeframe: Predose up to 12 hours postdose at Week 24Population: PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum.
Outcome measures
| Measure |
Ataluren
n=6 Participants
Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for up to 24 weeks.
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|---|---|
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Maximum Concentration (Cmax) of Ataluren
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12.6 μg/mL
Standard Deviation 3.64
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SECONDARY outcome
Timeframe: Predose up to 12 hours postdose at Week 24Population: PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum.
Outcome measures
| Measure |
Ataluren
n=6 Participants
Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for up to 24 weeks.
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|---|---|
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Time to Maximum Plasma Concentration (Tmax) of Ataluren
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2.08 hours
Interval 1.0 to 4.13
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SECONDARY outcome
Timeframe: Predose up to 12 hours postdose at Week 24Population: PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
Ataluren
n=5 Participants
Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for up to 24 weeks.
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|---|---|
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Trough Concentration (Ctrough) of Ataluren
|
3.48 μg/mL
Standard Deviation 3.36
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Adverse Events
Ataluren
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Ataluren
n=6 participants at risk
Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for up to 24 weeks.
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|---|---|
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Gastrointestinal disorders
Diarrhoea
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33.3%
2/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Gastrointestinal disorders
Teething
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Gastrointestinal disorders
Vomiting
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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General disorders
Pyrexia
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Infections and infestations
Body tinea
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Infections and infestations
Enterovirus infection
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Infections and infestations
Gastroenteritis
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33.3%
2/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Infections and infestations
Hand-foot-and-mouth disease
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
|
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Infections and infestations
Otitis media
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Infections and infestations
Upper respiratory tract infection
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Infections and infestations
Viral upper respiratory tract infection
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Injury, poisoning and procedural complications
Fall
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
|
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Metabolism and nutrition disorders
Decreased appetite
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Skin and subcutaneous tissue disorders
Eczema
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Skin and subcutaneous tissue disorders
Skin plaque
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16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Skin and subcutaneous tissue disorders
Urticaria
|
16.7%
1/6 • Baseline up to Week 28
Safety analysis set included all participants who received at least 1 dose of ataluren.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER