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

Conditions

Muscular Dystrophy, Duchenne

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.

Eugenio Mercuri, A. Nascimento Osorio, Francesco Muntoni, Filippo Buccella, Isabelle Desguerre et al. (17 authors)

Journal of Neurology2023-04-2841 citations

10.1007/s00415-023-11687-1

Ataluren Preserves Upper Limb Function in nmDMD Patients from Study 041, a Phase 3 Placebo-Controlled Trial, and the STRIDE Registry (S34.008)

Craig M. McDonald, Eugenio Mercuri, Francesco Muntoni, Heather Gordish‐Dressman, Lauren P. Morgenroth et al. (19 authors)

Neurology2023-04-251 citations

10.1212/wnl.0000000000202507

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

Eugenio Mercuri, Francesco Muntoni, A. Nascimento Osorio, M. Tulinius, Filippo Buccella et al. (17 authors)

Journal of Comparative Effectiveness Research2020-01-30124 citations

10.2217/cer-2019-0171

Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry

Francesco Muntoni, Isabelle Desguerre, Michela Guglieri, A. Nascimento Osorio, Janbernd Kirschner et al. (15 authors)

Journal of Comparative Effectiveness Research2019-08-1537 citations

10.2217/cer-2019-0086

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

Healthy volunteers

No

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

Design outcomes

Primary

Measure	Time frame
Percentage of Participants With Adverse Events	5 years

Secondary

Measure	Time frame
Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling	5 years

Countries

Austria, Brazil, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Latvia, Norway, Portugal, Romania, Slovenia, Sweden, United Kingdom

Contacts

STUDY_DIRECTORShelley Johnson

PTC Therapeutics

Outcome results