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Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

NCT01247207 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 270

Last updated 2026-03-09

No results posted yet for this study

Summary

The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial).

The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.

Conditions

Interventions

DRUG

Ataluren

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

Sponsors & Collaborators

Principal Investigators

  • Vinay Penematsa, MD · PTC Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-11-30
Primary Completion
2026-02-10
Completion
2026-02-10

Countries

  • United States
  • Canada

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 NCT01247207 on ClinicalTrials.gov