Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Active, not recruiting

• Conditions: Muscular Dystrophy, Duchenne

• Registration Number: NCT02369731
• Lead Sponsor: PTC Therapeutics

Brief Summary

This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety, effectiveness, and prescription patterns in routine clinical practice.

Detailed Description

This is a multicenter, observational study of participants receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 360 participants across \~50 care centers in Europe and other regions over a period of \~ 2 years. The study population will include participants who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program) and who provide consent. Participants will be followed for at least 5 years from their date of enrollment. Safety and efficacy data will be collected in conjunction with routine visits conducted as per usual care. Although there are no protocol-mandated procedures, it is expected that physicians and other caregivers will follow published treatment guidelines and standards of care.

Study Timeline

• Study First Submitted: 2015-01-28
• Study First Submitted QC: 2015-02-17
• Study First Posted: 2015-02-24
• Study Start: 2015-04-30
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-13
• Last Update Posted: 2025-03-14
• Primary Completion: 2025-05-30
• Study Completion: 2025-05-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 316

Inclusion Criteria

• Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program)
• Willing to provide written informed consent to allow the study data collection procedures (either by the participant or through authorisation by a legal guardian)

Exclusion Criteria

• Participants who are receiving ataluren or placebo in a blinded, randomized clinical trial, or ataluren in any other ataluren clinical trial or cohort early access program that prevents participation in this study

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With Adverse Events	5 years

Secondary Outcome Measures

Name	Time	Method
Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling	5 years

Trial Locations

Locations (71)

Krankenhaus der Barmherzigen Schwestern Linz; 🇦🇹 Linz, Austria

Sozialmedizinisches Zentrum Süd Kaiser-Franz-Josef Spital mit Gottfried von Preyer´schen Kinderspital; 🇦🇹 Wien, Austria

Hospital das Clínicas da Universidade de São Paulo; 🇧🇷 São Paulo, Brazil

The University Hospital Brno; 🇨🇿 Brno, Czechia

Motol University Hospital; 🇨🇿 Praha 5 - Motol, Czechia

CHU de Bordeaux Hôpital Pellegrin Enfant; 🇫🇷 Bordeaux Cedex, France

CHRU de Brest Hôpital Morvan; 🇫🇷 Brest, France

Hospices Civils de Lyon Groupement Hospitalier Est - Bâtiment "Les Tilleuls"; 🇫🇷 Bron Cedex, France

CHU de Clermont-Ferrand, hôpital Estaing; 🇫🇷 Clermont-Ferrand, France

CHU de Martinique - Hôpital P. Zobda-Quitman; 🇫🇷 Fort De France Cedex, France

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