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Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
study id #: NCT02369731
condition: Duchenne Muscular Dystrophy
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.
mechanism of action: Exon-skipping to promote dystrophin production
last updated: December 06, 2019
start date: April 2015
estimated completion: April 2022
size / enrollment: 200
This is a multicenter, observational study of patients receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 200 patients across ~50 care centers in Europe and other regions over a period of ~ 2 years. The study population will include patients who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named patient early access program) and who provide consent. Patients 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.
- Incidence of adverse events [ Time Frame: 5 years ]
- Changes in laboratory parameters [Time Frame: 5 years]
- Changes in blood pressure [Time Frame: 5 years]
- Prescriber and patient compliance with prescribing information according to the approved labelling [Time Frame: 5 years]
• Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named patient early access program)
• Willing to provide written informed consent to allow the study data collection procedures (either by the patient or through authorisation by a legal guardian)
• Patients 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
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