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)

Bērnu klīniskā universitātes slimnīca; 🇱🇻 Riga, Latvia

Karolinska Institutet; 🇸🇪 Stockholm, Sweden

Children Hospital - Children's Neurosciences Leeds TEACHING HOSPITALS NHS Trust; 🇬🇧 Leeds, United Kingdom

UCL Great Ormond Street Institute of Child Health; 🇬🇧 London, United Kingdom

Royal Manchester Children's Hospital; 🇬🇧 Manchester, United Kingdom

Ahepa University Hospital; 🇬🇷 Thessaloniki, Greece

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; 🇮🇹 Milano, Italy

ASST Grande Ospedale Metropolitano Niguarda; 🇮🇹 Milano, Italy

II Policlinico Neurofisiopatologia (Edificio 11 G); 🇮🇹 Napoli, Italy

Cardiomiologia e Genetica Medica Primo Policlinico; 🇮🇹 Napoli, Italy

Bambino Gesù Children's Hospital; 🇮🇹 Rome, Italy

Ospedale Molinette SCDU Neuropsichiatria Infantile AO Città della Salute e della Scienza di Torino; 🇮🇹 Torino, Italy

Centro Hospitalar Lisboa Norte EPE - Hospital de Santa Maria; 🇵🇹 Lisboa, Portugal

University Children's Hospital University Medical Centre Ljubljana; 🇸🇮 Ljubljana, Slovenia

Göteborgs Universitet Avdelningen för pediatrik vid institutionen för kliniska vetenskaper; 🇸🇪 Goteborg, Sweden

Birmingham Heartlands Hospital; 🇬🇧 Birmingham, United Kingdom

Children's Hospital Alder Hey Children's Hospital NHS Trust; 🇬🇧 Liverpool, United Kingdom

St Thomas' Hospital Children Services; 🇬🇧 London, United Kingdom

Bristol Children's Hospital; 🇬🇧 Bristol, United Kingdom

Addenbrooke's Hospital; 🇬🇧 Cambridge, United Kingdom

The John Walton Muscular Dystrophy Research Centre; 🇬🇧 Newcastle, United Kingdom

NIHR Lancashire Clinical Research Facility Royal Preston Hospital; 🇬🇧 Preston, United Kingdom

Hadassah Mount Scopus Medical Center; 🇮🇱 Jerusalem, Israel

Schneider Medical Center Tel Aviv University; 🇮🇱 Petah Tikva, Israel

CHU de Toulouse Hôpital Pierre Paul Riquet; 🇫🇷 Toulouse Cedex, France

The University Hospital Brno; 🇨🇿 Brno, Czechia

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

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

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

CHRU de Lille; 🇫🇷 Lille Cedex, France

CHU de Marseille Hôpital de la Timone; 🇫🇷 Marseille Cedex 5, France

CHU de Nantes; 🇫🇷 Nantes, France

Hôpital Armand Trousseau; 🇫🇷 Paris, France

Hôpital Necker - Enfants malades; 🇫🇷 Paris, France

CHU de Reims American Memorial Hospital; 🇫🇷 Reims Cedex, France

CHU de Saint-Etienne Hôpital Bellevue; 🇫🇷 Saint Etienne Cedex 2, France

CHU de Strasbourg; 🇫🇷 Strasbourg Cedex, France

CHU de la Réunion - GHSR - GH Sud-Réunion; 🇫🇷 Saint Pierre, France

CHRU de Tours Hôpital Bretonneau; 🇫🇷 Tours, France

CHU de Nancy Hôpital de Brabois-Enfants Rue du Morvan; 🇫🇷 Vandoeuvre-les-Nancy, France

Klinikum Bayreuth GmbH; 🇩🇪 Bayreuth, Germany

Sozialpädiatrisches Zentrum (SPZ) Charité Berlin; 🇩🇪 Berlin, Germany

Universitätsklinikum Erlangen; 🇩🇪 Erlangen, Germany

SPZ Frankfurt Mitte; 🇩🇪 Frankfurt am Main, Germany

Universitaetsklinikum Essen (AoR); 🇩🇪 Essen, Germany

Universitaetsklinikum Freiburg; 🇩🇪 Freiburg, Germany

MVZ Kinder- und Jugendärzte Fürth GmbH; 🇩🇪 Fürth, Germany

Universitätskinderklinik Gießen; 🇩🇪 Gießen, Germany

Klinik und Poliklinik f. Kinder- und Jugendmedizin; 🇩🇪 Hamburg, Germany

Muskelzentrum, Schwerpunkt Neuropädiatrie; 🇩🇪 Kassel, Germany

Children's Hospital "Aghia Sophia"; 🇬🇷 Athens, Greece

Semmelweis Egyetem II (Semmelweis University II); 🇭🇺 Budapest, Hungary

IRCSS Istituto delle Scienze Neurologiche; 🇮🇹 Bologna, Italy

Szent László Kórház Gyermekneurológiai szakrendelés; 🇭🇺 Budapest, Hungary

Sheba Medical Center The Joseph Sagol EMG, Neuroscience Center; 🇮🇱 Ramat Gan, Israel

Istituto Giannina Gaslini; 🇮🇹 Genova, Italy

AOU Policlinico "G. Martino"; 🇮🇹 Messina, Italy

Fondazione Policlinico Universitario Agostino Gemelli; 🇮🇹 Roma, Italy

IRCCS Istituto Neurologico Carlo Besta; 🇮🇹 Milano, Italy

AO Padova Clinica Neurologica; 🇮🇹 Padova, Italy

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

Krankenhaus der Barmherzigen Schwestern Linz; 🇦🇹 Linz, Austria

St. Olavs hospital University children's clinical hospital; 🇳🇴 Trondheim, Norway

Centro Hospitalar do Porto EPE - Hospital Geral de Santo António; 🇵🇹 Porto, Portugal

Spitalul Clinic de Copii "Dr. Victor Gomoiu" Sectia Clinica Neurologie Pediatrica; 🇷🇴 Bucharest, Romania

Centro Hospitalar e Universitário de coimbra EPE - Hospital Pediátrico de Coimbra; 🇵🇹 Coimbra, Portugal

Regina Maria Policlinica Opera Center; 🇷🇴 Bucharest, Romania