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

Plain-language summary

This study evaluates the drug Ataluren in boys aged 2 to under 5 years with Duchenne muscular dystrophy who carry a nonsense mutation in the dystrophin gene (DMD). It enrolled young boys weighing at least 12 kg with a diagnosis of DMD. It was conducted in Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Completed

Sponsor

PTC Therapeutics

Intervention

Ataluren

Start date

6/9/2016

Estimated completion

2/9/2018

Countries

United States

Related genes

Locations

• Gulf Breeze, United States
• Chicago, United States
• Boston, United States
• Cincinnati, United States
• Dallas, United States
• Salt Lake City, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Males ≥2 to \<5 years of age
* Body weight ≥12 kg
* Diagnosis of DMD
* Nonsense mutation in at least 1 allele of the dystrophin gene

Exclusion Criteria:

* Participation in any other drug or device clinical investigation
* Ongoing use of prohibited concomitant medications