welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
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
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
Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyThis study is a long-term study of atalu...
Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular DystrophyDystrophinopathy is a disease continuum ...
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin geneThis guidance is not intended to affect ...
New Analyses of Translarna™ (ataluren) Data from ACT DMD Presented at the 12th Annual European Pediatric Neurology...PTC Therapeutics, Inc. today announced t...
A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged 2 to 5 years with nonsense mutation ...Nonsense mutation Duchenne muscular dyst...
PTC Therapeutics Phase III Duchenne Muscular Dystrophy (DMD) Clinical TrialPTC Therapeutics, Inc. is seeking pati...
Safety and Tolerability of Ataluren in a Phase 3 Study of Patients with Nonsense Mutation Duchenne Muscular Dystroph...Objective: Examine the safety/tolerabil...