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

NCT00592553 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 174

Last updated 2020-04-07

Study results available
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Summary

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 13 percent (%) 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 2b trial that will evaluate the clinical benefit of ataluren in boys with DMD/BMD due to a nonsense mutation. The main goals of the study are to understand whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time.

Conditions

Interventions

DRUG

Ataluren

Ataluren will be administered as per the dose and schedule specified in the respective arms.

DRUG

Placebo

Placebo matching to ataluren will be administered as the schedule specified in the respective arm.

Sponsors & Collaborators

Principal Investigators

  • Leone Atkinson, MD, PhD · PTC Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
5 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-02-29
Primary Completion
2009-12-31
Completion
2009-12-31

Countries

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

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 NCT00592553 on ClinicalTrials.gov