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

NCT ID: NCT02369731

Last Updated: 2025-03-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

316 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-04-30

Study Completion Date

2025-05-30

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Muscular Dystrophy, Duchenne

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

No interventions assigned to this group

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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The John Walton Muscular Dystrophy Research Centre (TREAT-NMD)

UNKNOWN

Sponsor Role collaborator

Cooperative International Neuromuscular Research Group

NETWORK

Sponsor Role collaborator

PTC Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Shelley Johnson

Role: STUDY_DIRECTOR

PTC Therapeutics

Locations

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CHU de la Réunion - GHSR - GH Sud-Réunion

Saint-Pierre, , France

Site Status

CHU de Strasbourg

Strasbourg, , France

Site Status

Krankenhaus der Barmherzigen Schwestern Linz

Linz, , Austria

Site Status

Sozialmedizinisches Zentrum Süd Kaiser-Franz-Josef Spital mit Gottfried von Preyer´schen Kinderspital

Vienna, , Austria

Site Status

Hospital das Clínicas da Universidade de São Paulo

São Paulo, , Brazil

Site Status

The University Hospital Brno

Brno, , Czechia

Site Status

Motol University Hospital

Praha 5 - Motol, , Czechia

Site Status

CHU de Bordeaux Hôpital Pellegrin Enfant

Bordeaux, , France

Site Status

CHRU de Brest Hôpital Morvan

Brest, , France

Site Status

Hospices Civils de Lyon Groupement Hospitalier Est - Bâtiment "Les Tilleuls"

Bron, , France

Site Status

CHU de Clermont-Ferrand, hôpital Estaing

Clermont-Ferrand, , France

Site Status

CHU de Martinique - Hôpital P. Zobda-Quitman

Fort de France Cedex, , France

Site Status

CHRU de Lille

Lille, , France

Site Status

CHU de Marseille Hôpital de la Timone

Marseille, , France

Site Status

CHU de Nantes

Nantes, , France

Site Status

Hôpital Armand Trousseau

Paris, , France

Site Status

Hôpital Necker - Enfants malades

Paris, , France

Site Status

CHU de Reims American Memorial Hospital

Reims, , France

Site Status

CHU de Saint-Etienne Hôpital Bellevue

Saint-Etienne, , France

Site Status

CHU de Toulouse Hôpital Pierre Paul Riquet

Toulouse, , France

Site Status

CHRU de Tours Hôpital Bretonneau

Tours, , France

Site Status

CHU de Nancy Hôpital de Brabois-Enfants Rue du Morvan

Vandœuvre-lès-Nancy, , France

Site Status

Klinikum Bayreuth GmbH

Bayreuth, , Germany

Site Status

Sozialpädiatrisches Zentrum (SPZ) Charité Berlin

Berlin, , Germany

Site Status

Universitätsklinikum Erlangen

Erlangen, , Germany

Site Status

Universitaetsklinikum Essen (AoR)

Essen, , Germany

Site Status

SPZ Frankfurt Mitte

Frankfurt am Main, , Germany

Site Status

Universitaetsklinikum Freiburg

Freiburg im Breisgau, , Germany

Site Status

MVZ Kinder- und Jugendärzte Fürth GmbH

Fürth, , Germany

Site Status

Universitätskinderklinik Gießen

Giessen, , Germany

Site Status

Klinik und Poliklinik f. Kinder- und Jugendmedizin

Hamburg, , Germany

Site Status

Muskelzentrum, Schwerpunkt Neuropädiatrie

Kassel, , Germany

Site Status

Children's Hospital "Aghia Sophia"

Athens, , Greece

Site Status

Ahepa University Hospital

Thessaloniki, , Greece

Site Status

Semmelweis Egyetem II (Semmelweis University II)

Budapest, , Hungary

Site Status

Szent László Kórház Gyermekneurológiai szakrendelés

Budapest, , Hungary

Site Status

Hadassah Mount Scopus Medical Center

Jerusalem, , Israel

Site Status

Schneider Medical Center Tel Aviv University

Petah Tikva, , Israel

Site Status

Sheba Medical Center The Joseph Sagol EMG, Neuroscience Center

Ramat Gan, , Israel

Site Status

IRCSS Istituto delle Scienze Neurologiche

Bologna, , Italy

Site Status

Istituto Giannina Gaslini

Genova, , Italy

Site Status

AOU Policlinico "G. Martino"

Messina, , Italy

Site Status

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

IRCCS Istituto Neurologico Carlo Besta

Milan, , Italy

Site Status

ASST Grande Ospedale Metropolitano Niguarda

Milan, , Italy

Site Status

II Policlinico Neurofisiopatologia (Edificio 11 G)

Napoli, , Italy

Site Status

Cardiomiologia e Genetica Medica Primo Policlinico

Napoli, , Italy

Site Status

AO Padova Clinica Neurologica

Padua, , Italy

Site Status

Fondazione Policlinico Universitario Agostino Gemelli

Roma, , Italy

Site Status

Bambino Gesù Children's Hospital

Rome, , Italy

Site Status

Ospedale Molinette SCDU Neuropsichiatria Infantile AO Città della Salute e della Scienza di Torino

Torino, , Italy

Site Status

Bērnu klīniskā universitātes slimnīca

Riga, , Latvia

Site Status

St. Olavs hospital University children's clinical hospital

Trondheim, , Norway

Site Status

Centro Hospitalar e Universitário de coimbra EPE - Hospital Pediátrico de Coimbra

Coimbra, , Portugal

Site Status

Centro Hospitalar Lisboa Norte EPE - Hospital de Santa Maria

Lisbon, , Portugal

Site Status

Centro Hospitalar do Porto EPE - Hospital Geral de Santo António

Porto, , Portugal

Site Status

Spitalul Clinic de Copii "Dr. Victor Gomoiu" Sectia Clinica Neurologie Pediatrica

Bucharest, , Romania

Site Status

Regina Maria Policlinica Opera Center

Bucharest, , Romania

Site Status

University Children's Hospital University Medical Centre Ljubljana

Ljubljana, , Slovenia

Site Status

Göteborgs Universitet Avdelningen för pediatrik vid institutionen för kliniska vetenskaper

Gothenburg, , Sweden

Site Status

Karolinska Institutet

Stockholm, , Sweden

Site Status

Birmingham Heartlands Hospital

Birmingham, , United Kingdom

Site Status

Bristol Children's Hospital

Bristol, , United Kingdom

Site Status

Addenbrooke's Hospital

Cambridge, , United Kingdom

Site Status

Children Hospital - Children's Neurosciences Leeds TEACHING HOSPITALS NHS Trust

Leeds, , United Kingdom

Site Status

Children's Hospital Alder Hey Children's Hospital NHS Trust

Liverpool, , United Kingdom

Site Status

St Thomas' Hospital Children Services

London, , United Kingdom

Site Status

UCL Great Ormond Street Institute of Child Health

London, , United Kingdom

Site Status

Royal Manchester Children's Hospital

Manchester, , United Kingdom

Site Status

The John Walton Muscular Dystrophy Research Centre

Newcastle, , United Kingdom

Site Status

NIHR Lancashire Clinical Research Facility Royal Preston Hospital

Preston, , United Kingdom

Site Status

Countries

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

References

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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.

Reference Type DERIVED
PMID: 37115359 (View on PubMed)

Mercuri 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.

Reference Type DERIVED
PMID: 31997646 (View on PubMed)

Other Identifiers

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PTC124-GD-025o-DMD

Identifier Type: -

Identifier Source: org_study_id

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