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Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

NCT00264888 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 38

Last updated 2009-01-14

No results posted yet for this study

Summary

In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein. PTC124 has been shown to partially restore dystrophin production in animals with DMD due to a nonsense mutation. The main purpose of this study is to understand whether PTC124 can safely increase functional dystrophin protein in the muscles of patients with DMD due to a nonsense mutation.

Conditions

Interventions

DRUG

PTC124

Sponsors & Collaborators

Principal Investigators

  • Richard Finkel, MD · Children's Hospital of Philadelphia

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-12-31
Primary Completion
2007-05-31
Completion
2007-05-31

Countries

  • United States

Study Locations

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Entities

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 NCT00264888 on ClinicalTrials.gov