This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to \<2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).
Participants who complete the 24-week treatment period in this study will be offered participation to a follow-up extension period for at least 52 weeks from the date of first administration of ataluren in this parent study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
Ataluren will be administered as per the dose and schedule specified in the arm.
Rare Disease Research, LLC
Atlanta, Georgia, United States
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
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.
Time frame: Baseline up to Week 28
Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren
Time frame: Predose up to 12 hours postdose at Week 24
Area Under the Concentration-Time Curve Between Dosing Interval (AUC0-τ) of Ataluren
Time frame: Predose up to 12 hours postdose at Week 24
Maximum Concentration (Cmax) of Ataluren
Time frame: Predose up to 12 hours postdose at Week 24
Time to Maximum Plasma Concentration (Tmax) of Ataluren
Time frame: Predose up to 12 hours postdose at Week 24
Trough Concentration (Ctrough) of Ataluren
Time frame: Predose up to 12 hours postdose at Week 24
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