NCT02819557

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: Male
• Age: 2 Years – 5 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to \<5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.

Detailed description

In nonsense mutation DMD (nmDMD), early start of treatment is important and necessary and, therefore, it is relevant to understand the correct and tolerable dose in this age group, particularly since ataluren is dosed by weight. This study included a 4-week screening period, a 52-week treatment period (the first 4 weeks of which included PK parameters), and a 4-week follow-up period for participants who completed the treatment period (60 weeks total duration). The objective of the extension period (treatment period after PK parameters have been completed) was to assess the long-term safety of chronic administration of ataluren in this participant population.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Ataluren	White to off-white powder for oral suspension.

Timeline

• Start date: 2016-06-09
• Primary completion: 2018-02-09
• Completion: 2018-02-09
• First posted: 2016-06-30
• Last updated: 2020-08-28
• Results posted: 2020-08-28

Locations

6 sites across 1 country: United States