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Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT01826487 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 230

Last updated 2020-08-04

Study results available
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Summary

Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which 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. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy in approximately 10-15 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. The main goal of this Phase 3 study is to evaluate the effect of ataluren on walking ability. The effect of ataluren on physical function, quality of life, and activities of daily living will be evaluated. This study will also provide additional information on the long-term safety of ataluren.

Conditions

  • Muscular Dystrophy, Duchenne
  • Muscular Dystrophies
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Musculoskeletal Diseases
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Genetic Diseases, X-Linked
  • Genetic Diseases, Inborn

Interventions

DRUG

Ataluren

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

DRUG

Placebo

Placebo will be administered as per the schedule specified in the arm.

Sponsors & Collaborators

Principal Investigators

  • Robert Spiegel, M.D. · PTC Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
7 Years
Max Age
16 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-03-26
Primary Completion
2015-08-20
Completion
2015-08-20

Countries

  • United States
  • Australia
  • Belgium
  • Brazil
  • Canada
  • Chile
  • Czechia
  • France
  • Germany
  • Israel
  • Italy
  • Poland
  • South Korea
  • Spain
  • Sweden
  • Switzerland
  • Turkey (Türkiye)
  • 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 NCT01826487 on ClinicalTrials.gov