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)

An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: Male
• Age: 6 Months – 2 Years
• Healthy volunteers: Not accepted

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).

Detailed description

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.

Conditions

• Nonsene Mutation Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Ataluren	Ataluren will be administered as per the dose and schedule specified in the arm.

Timeline

• Start date: 2021-12-29
• Primary completion: 2023-08-07
• Completion: 2023-08-07
• First posted: 2020-04-07
• Last updated: 2024-04-01
• Results posted: 2024-04-01

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study