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Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00847379 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 173

Last updated 2020-07-15

Study results available
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Summary

Duchenne/Becker muscular dystrophy (DMD/BMD) 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/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2b extension trial that will evaluate the long-term safety of ataluren (PTC124) in boys with nonsense mutation DMD/BMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, and other important clinical and laboratory measures.

Conditions

Interventions

DRUG

Ataluren

Ataluren oral powder for suspension will be administered as per dose and schedule specified in the arm.

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    collaborator INDUSTRY

  • PTC Therapeutics

    lead INDUSTRY

Principal Investigators

  • Leone Atkinson, M.D., Ph.D. · PTC Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-01-31
Primary Completion
2010-05-24
Completion
2010-05-24

Countries

  • United States
  • Australia
  • Belgium
  • Canada
  • France
  • Germany
  • Israel
  • Italy
  • Spain
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

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 NCT00847379 on ClinicalTrials.gov