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Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

NCT00759876 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2020-10-29

Study results available
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Summary

Duchenne muscular dystrophy (DMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of DMD in approximately 10-15% of boys with the disease. Ataluren is an orally-delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a extension trial that will evaluate the long-term safety of ataluren in boys with nonsense mutation DMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, strength, and other important clinical and laboratory measures.

Conditions

Interventions

DRUG

Ataluren

Ataluren will be provided as a vanilla-flavored powder to be mixed with milk. Dosing based on participant body weight

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    collaborator INDUSTRY

  • PTC Therapeutics

    lead INDUSTRY

Principal Investigators

  • Leone Atkinson · PTC Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-08-13
Primary Completion
2010-05-17
Completion
2010-05-17

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00759876 on ClinicalTrials.gov