Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care
1 other identifier
observational
316
16 countries
71
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Apr 2015
Longer than P75 for all trials
71 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 28, 2015
CompletedFirst Posted
Study publicly available on registry
February 24, 2015
CompletedStudy Start
First participant enrolled
April 30, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
March 31, 2025
CompletedFebruary 24, 2026
April 1, 2025
9.9 years
January 28, 2015
February 20, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Percentage of Participants With Adverse Events
5 years
Secondary Outcomes (1)
Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling
5 years
Other Outcomes (1)
Participant Health Management Measures
5 years
Study Arms (1)
Translarna
Participants with nmDMD receiving Translarna will be followed for at least 5 years from their date of enrollment, or until participant withdrawal of consent or death, whichever occurs first.
Eligibility Criteria
The target study population will include participants for whom the decision to prescribe Translarna (outside of a clinical trial) has already been made, including participants in named participant early access programs. Participants within each prescriber's practice who are or will be receiving treatment with Translarna, and who meet the eligibility criteria and provide informed consent (either by the participant or through authorisation by a legal guardian) will be invited to enroll into the study and will be followed according to the protocol.
You may qualify if:
- 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)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (71)
Krankenhaus der Barmherzigen Schwestern Linz
Linz, 4010, Austria
Sozialmedizinisches Zentrum Süd Kaiser-Franz-Josef Spital mit Gottfried von Preyer´schen Kinderspital
Vienna, 1100, Austria
Hospital das Clínicas da Universidade de São Paulo
São Paulo, 05403-000, Brazil
The University Hospital Brno
Brno, 62500, Czechia
Motol University Hospital
Praha 5 - Motol, 15006, Czechia
CHU de Bordeaux Hôpital Pellegrin Enfant
Bordeaux, 33076, France
CHRU de Brest Hôpital Morvan
Brest, 29200, France
Hospices Civils de Lyon Groupement Hospitalier Est - Bâtiment "Les Tilleuls"
Bron, 69677, France
CHU de Clermont-Ferrand, hôpital Estaing
Clermont-Ferrand, 63003, France
CHU de Martinique - Hôpital P. Zobda-Quitman
Fort de France Cedex, 97261, France
CHRU de Lille
Lille, 59037, France
CHU de Marseille Hôpital de la Timone
Marseille, 13385, France
CHU de Nantes
Nantes, 44093, France
Hôpital Armand Trousseau
Paris, 75012, France
Hôpital Necker - Enfants malades
Paris, 75015, France
CHU de Reims American Memorial Hospital
Reims, 51092, France
CHU de Saint-Etienne Hôpital Bellevue
Saint-Etienne, 42055, France
CHU de la Réunion - GHSR - GH Sud-Réunion
Saint-Pierre, 97448, France
CHU de Strasbourg
Strasbourg, 67098, France
CHU de Toulouse Hôpital Pierre Paul Riquet
Toulouse, 31059, France
CHRU de Tours Hôpital Bretonneau
Tours, 37044, France
CHU de Nancy Hôpital de Brabois-Enfants Rue du Morvan
Vandœuvre-lès-Nancy, 54400, France
Klinikum Bayreuth GmbH
Bayreuth, 95445, Germany
Sozialpädiatrisches Zentrum (SPZ) Charité Berlin
Berlin, 13353, Germany
Universitätsklinikum Erlangen
Erlangen, 91054, Germany
Universitaetsklinikum Essen (AoR)
Essen, 45122, Germany
SPZ Frankfurt Mitte
Frankfurt am Main, 60316, Germany
Universitaetsklinikum Freiburg
Freiburg im Breisgau, 79106, Germany
MVZ Kinder- und Jugendärzte Fürth GmbH
Fürth, 90762, Germany
Universitätskinderklinik Gießen
Giessen, 35385, Germany
Klinik und Poliklinik f. Kinder- und Jugendmedizin
Hamburg, 20246, Germany
Muskelzentrum, Schwerpunkt Neuropädiatrie
Kassel, 34125, Germany
Children's Hospital "Aghia Sophia"
Athens, 11527, Greece
Ahepa University Hospital
Thessaloniki, 54621, Greece
Semmelweis Egyetem II (Semmelweis University II)
Budapest, 01094, Hungary
Szent László Kórház Gyermekneurológiai szakrendelés
Budapest, Hungary
Hadassah Mount Scopus Medical Center
Jerusalem, 91240, Israel
Schneider Medical Center Tel Aviv University
Petah Tikva, 49202, Israel
Sheba Medical Center The Joseph Sagol EMG, Neuroscience Center
Ramat Gan, 52621, Israel
IRCSS Istituto delle Scienze Neurologiche
Bologna, 40133, Italy
Istituto Giannina Gaslini
Genova, 16147, Italy
AOU Policlinico "G. Martino"
Messina, 98125, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
IRCCS Istituto Neurologico Carlo Besta
Milan, 20133, Italy
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162, Italy
II Policlinico Neurofisiopatologia (Edificio 11 G)
Naples, 80131, Italy
Cardiomiologia e Genetica Medica Primo Policlinico
Naples, 80138, Italy
AO Padova Clinica Neurologica
Padua, 35128, Italy
Fondazione Policlinico Universitario Agostino Gemelli
Roma, 00168, Italy
Bambino Gesù Children's Hospital
Rome, 00146, Italy
Ospedale Molinette SCDU Neuropsichiatria Infantile AO Città della Salute e della Scienza di Torino
Torino, 10126, Italy
Bērnu klīniskā universitātes slimnīca
Riga, Latvia
St. Olavs hospital University children's clinical hospital
Trondheim, NO-7006, Norway
Centro Hospitalar e Universitário de coimbra EPE - Hospital Pediátrico de Coimbra
Coimbra, 3000-075, Portugal
Centro Hospitalar Lisboa Norte EPE - Hospital de Santa Maria
Lisbon, 1649-035, Portugal
Centro Hospitalar do Porto EPE - Hospital Geral de Santo António
Porto, 4099-001, Portugal
Spitalul Clinic de Copii "Dr. Victor Gomoiu" Sectia Clinica Neurologie Pediatrica
Bucharest, 22113, Romania
Regina Maria Policlinica Opera Center
Bucharest, Romania
University Children's Hospital University Medical Centre Ljubljana
Ljubljana, 1000, Slovenia
Göteborgs Universitet Avdelningen för pediatrik vid institutionen för kliniska vetenskaper
Gothenburg, 41685, Sweden
Karolinska Institutet
Stockholm, 17176, Sweden
Birmingham Heartlands Hospital
Birmingham, B9 5SS, United Kingdom
Bristol Children's Hospital
Bristol, BS2 8BJ, United Kingdom
Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
Children Hospital - Children's Neurosciences Leeds TEACHING HOSPITALS NHS Trust
Leeds, LS1 3EX, United Kingdom
Children's Hospital Alder Hey Children's Hospital NHS Trust
Liverpool, L12 2AP, United Kingdom
St Thomas' Hospital Children Services
London, SE1 7EH, United Kingdom
UCL Great Ormond Street Institute of Child Health
London, WC1N 1EH, United Kingdom
Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
The John Walton Muscular Dystrophy Research Centre
Newcastle, NE1 3BZ, United Kingdom
NIHR Lancashire Clinical Research Facility Royal Preston Hospital
Preston, PR2 9HT, United Kingdom
Related Publications (2)
Mercuri E, Osorio AN, Muntoni F, Buccella F, Desguerre I, Kirschner J, Tulinius M, de Resende MBD, Morgenroth LP, Gordish-Dressman H, Johnson S, Kristensen A, Werner C, Trifillis P, Henricson EK, McDonald CM; STRIDE and CINRG DNHS investigators. Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis. J Neurol. 2023 Aug;270(8):3896-3913. doi: 10.1007/s00415-023-11687-1. Epub 2023 Apr 28.
PMID: 37115359DERIVEDMercuri E, Muntoni F, Osorio AN, Tulinius M, Buccella F, Morgenroth LP, Gordish-Dressman H, Jiang J, Trifillis P, Zhu J, Kristensen A, Santos CL, Henricson EK, McDonald CM, Desguerre I; STRIDE; CINRG Duchenne Natural History Investigators. Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. J Comp Eff Res. 2020 Apr;9(5):341-360. doi: 10.2217/cer-2019-0171. Epub 2020 Jan 30.
PMID: 31997646DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Shelley Johnson
PTC Therapeutics
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Target Duration
- 5 Years
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 28, 2015
First Posted
February 24, 2015
Study Start
April 30, 2015
Primary Completion
March 31, 2025
Study Completion
March 31, 2025
Last Updated
February 24, 2026
Record last verified: 2025-04