NCT04336826View on ClinicalTrials.gov

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)

PHASE2CompletedINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

December 29, 2021

Primary Completion Date

August 7, 2023

Study Completion Date

August 7, 2023

Conditions
Nonsene Mutation Duchenne Muscular Dystrophy
Interventions
DRUG

Ataluren

Ataluren will be administered as per the dose and schedule specified in the arm.

Trial Locations (1)

30329

Rare Disease Research, LLC, Atlanta

Sponsors

Lead Sponsor

PTC Therapeutics
All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY

NCT04336826 - 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) | Biotech Hunter | Biotech Hunter