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An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

NCT02090959 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 219

Last updated 2020-08-11

Study results available
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Summary

The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren \[PTC124-GD-020-DMD {NCT01826487}\]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study.

This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.

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 per the dose and schedule specified in the arm.

Sponsors & Collaborators

Principal Investigators

  • Francesco Bibbiani, M.D. · PTC Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2014-03-20
Primary Completion
2018-06-12
Completion
2018-06-12

Countries

  • United States
  • Australia
  • Belgium
  • Brazil
  • Bulgaria
  • Canada
  • Chile
  • Czechia
  • France
  • Germany
  • Israel
  • Italy
  • Poland
  • South Korea
  • Spain
  • Sweden
  • Switzerland
  • Turkey (Türkiye)
  • 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 NCT02090959 on ClinicalTrials.gov