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drugset / Trial / 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)

NCT04336826 ↗

Phase 2 Completed 6 enrolled PTC Therapeutics
NaSingle-groupOpen-labelTreatment

Summary

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).

Timeline

Start
2021-12-29
Primary completion
2023-08-07
Completion
2023-08-07

Drugs

EvaluationDrugModalityDoseRoute
Subject Ataluren Small molecule 10 mg/kg Oral
Subject Ataluren Small molecule 20 mg/kg Oral

Indications

Duchenne muscular dystrophy