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Completed

NCT Number: NCT02369731

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

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.

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Key information

Conditions

Muscular Dystrophy, Duchenne Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Krankenhaus der Barmherzigen Schwestern Linz, Linz, Austria

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Percentage of Participants With Adverse Events

    Time frame: 5 years

Secondary outcomes

  1. Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling

    Time frame: 5 years

Other outcomes

  1. Participant Health Management Measures

    Time frame: 5 years

Sponsors and collaborators

Lead sponsor

PTC Therapeutics

Industry

Collaborators

  • Cooperative International Neuromuscular Research Group
  • The John Walton Muscular Dystrophy Research Centre (TREAT-NMD)

Registry information

Official study title

Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care

Important dates

Study start
2015
Primary completion
2025
Study completion
2025
First posted
Feb 24, 2015
Registry last updated
Feb 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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