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Nonsense Mutation Readthrough Agent

Ataluren for Duchenne Muscular Dystrophy

Phase 3
Waitlist Available
Research Sponsored by PTC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to end of study (up to approximately 8 years)
Awards & highlights

Study Summary

This trial is for people with nmDBMD who have had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants. The purpose is to see if the treatment is safe and tolerated.

Who is the study for?
This trial is for individuals with a type of muscular dystrophy called nmDBMD who have previously taken Ataluren in a PTC sponsored clinical trial or treatment plan. It's also open to siblings of these participants if they've finished the placebo part of another study. Participants must understand and agree to the study requirements, be able to attend all visits, and men must use birth control during the study.Check my eligibility
What is being tested?
The trial is testing the safety and tolerability of Ataluren at doses of 10 mg/kg (twice daily) and 20 mg/kg (once daily). The medication will be given until certain conditions are met such as consent withdrawal, worsening condition after starting treatment, eligibility for another Ataluren trial, or commercial availability.See study design
What are the potential side effects?
While specific side effects aren't listed here, common ones from medications like Ataluren could include gastrointestinal discomforts like nausea or diarrhea, potential liver enzyme changes which would be monitored by blood tests, possible allergic reactions if there's known hypersensitivity to its ingredients.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to end of study (up to approximately 8 years)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to end of study (up to approximately 8 years) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants With Abnormal Physical Findings
Number of Participants With Adverse Events
Number of Participants With Laboratory Parameters Abnormalities

Side effects data

From 2018 Phase 4 trial • 1 Patients • NCT03256968
100%
increased cough
100%
80%
60%
40%
20%
0%
Study treatment Arm
Ataluren Administration

Trial Design

1Treatment groups
Experimental Treatment
Group I: AtalurenExperimental Treatment1 Intervention
Participants will receive 3 doses of ataluren oral suspension per day (10 mg/kg in the morning, 10 mg/kg at mid-day and 20 mg/kg in the evening).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ataluren
2018
Completed Phase 4
~1450

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
74 Previous Clinical Trials
5,960 Total Patients Enrolled
Vinay Penematsa, MDStudy DirectorPTC Therapeutics, Inc.
3 Previous Clinical Trials
520 Total Patients Enrolled

Media Library

Ataluren (Nonsense Mutation Readthrough Agent) Clinical Trial Eligibility Overview. Trial Name: NCT01247207 — Phase 3
Duchenne Muscular Dystrophy Research Study Groups: Ataluren
Duchenne Muscular Dystrophy Clinical Trial 2023: Ataluren Highlights & Side Effects. Trial Name: NCT01247207 — Phase 3
Ataluren (Nonsense Mutation Readthrough Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01247207 — Phase 3
Duchenne Muscular Dystrophy Patient Testimony for trial: Trial Name: NCT01247207 — Phase 3
~19 spots leftby Jun 2025
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