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

Muscular Dystrophy, Duchenne Clinical Trial

Official title:

Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02369731
• Other study ID #: PTC124-GD-025o-DMD
• Status: Active, not recruiting
• Start date: April 30, 2015
• Est. completion date: May 30, 2025

Study information

• Verified date: June 2024
• Source: PTC Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 316
• Est. completion date: May 30, 2025
• Est. primary completion date: May 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Locations

Country	Name	City	State
Austria	Krankenhaus der Barmherzigen Schwestern Linz	Linz
Austria	Sozialmedizinisches Zentrum Süd Kaiser-Franz-Josef Spital mit Gottfried von Preyer´schen Kinderspital	Wien
Brazil	Hospital das Clínicas da Universidade de São Paulo	São Paulo
Czechia	The University Hospital Brno	Brno
Czechia	Motol University Hospital	Praha 5 - Motol
France	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 Clermont-Ferrand, hôpital Estaing	Clermont-Ferrand
France	CHU de Martinique - Hôpital P. Zobda-Quitman	Fort De France Cedex
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 la Réunion - GHSR - GH Sud-Réunion	Saint Pierre
France	CHU de Strasbourg	Strasbourg Cedex
France	CHU de Toulouse Hôpital Pierre Paul Riquet	Toulouse Cedex
France	CHRU de Tours Hôpital Bretonneau	Tours
France	CHU de Nancy Hôpital de Brabois-Enfants Rue du Morvan	Vandoeuvre-les-Nancy
Germany	Klinikum Bayreuth GmbH	Bayreuth
Germany	Sozialpädiatrisches Zentrum (SPZ) Charité Berlin	Berlin
Germany	Universitätsklinikum Erlangen	Erlangen
Germany	Universitaetsklinikum Essen (AoR)	Essen
Germany	SPZ Frankfurt Mitte	Frankfurt am Main
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
Greece	Children's Hospital "Aghia Sophia"	Athens
Greece	Ahepa University Hospital	Thessaloniki
Hungary	Semmelweis Egyetem II (Semmelweis University II)	Budapest
Hungary	Szent László Kórház Gyermekneurológiai szakrendelés	Budapest
Israel	Hadassah Mount Scopus Medical Center	Jerusalem
Israel	Schneider Medical Center Tel Aviv University	Petah Tikva
Israel	Sheba Medical Center The Joseph Sagol EMG, Neuroscience Center	Ramat Gan
Italy	IRCSS Istituto delle Scienze Neurologiche	Bologna
Italy	Istituto Giannina Gaslini	Genova
Italy	AOU Policlinico "G. Martino"	Messina
Italy	ASST Grande Ospedale Metropolitano Niguarda	Milano
Italy	Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	Milano
Italy	IRCCS Istituto Neurologico Carlo Besta	Milano
Italy	Cardiomiologia e Genetica Medica Primo Policlinico	Napoli
Italy	II Policlinico Neurofisiopatologia (Edificio 11 G)	Napoli
Italy	AO Padova Clinica Neurologica	Padova
Italy	Fondazione Policlinico Universitario Agostino Gemelli	Roma
Italy	Bambino Gesù Children's Hospital	Rome
Italy	Ospedale Molinette SCDU Neuropsichiatria Infantile AO Città della Salute e della Scienza di Torino	Torino
Latvia	Bernu kliniska universitates slimnica	Riga
Norway	St. Olavs hospital University children's clinical hospital	Trondheim
Portugal	Centro Hospitalar e Universitário de coimbra EPE - Hospital Pediátrico de Coimbra	Coimbra
Portugal	Centro Hospitalar Lisboa Norte EPE - Hospital de Santa Maria	Lisboa
Portugal	Centro Hospitalar do Porto EPE - Hospital Geral de Santo António	Porto
Romania	Regina Maria Policlinica Opera Center	Bucharest
Romania	Spitalul Clinic de Copii "Dr. Victor Gomoiu" Sectia Clinica Neurologie Pediatrica	Bucharest
Slovenia	University Children's Hospital University Medical Centre Ljubljana	Ljubljana
Sweden	Göteborgs Universitet Avdelningen för pediatrik vid institutionen för kliniska vetenskaper	Goteborg
Sweden	Karolinska Institutet	Stockholm
United Kingdom	Birmingham Heartlands Hospital	Birmingham
United Kingdom	Bristol Children's Hospital	Bristol
United Kingdom	Addenbrooke's Hospital	Cambridge
United Kingdom	Children Hospital - Children's Neurosciences Leeds TEACHING HOSPITALS NHS Trust	Leeds
United Kingdom	Children's Hospital Alder Hey Children's Hospital NHS Trust	Liverpool
United Kingdom	St Thomas' Hospital Children Services	London
United Kingdom	UCL Great Ormond Street Institute of Child Health	London
United Kingdom	Royal Manchester Children's Hospital	Manchester
United Kingdom	The John Walton Muscular Dystrophy Research Centre	Newcastle
United Kingdom	NIHR Lancashire Clinical Research Facility Royal Preston Hospital	Preston

Sponsors (3)

Lead Sponsor	Collaborator
PTC Therapeutics	Cooperative International Neuromuscular Research Group, The John Walton Muscular Dystrophy Research Centre (TREAT-NMD)

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Participant Health Management Measures		5 years
Primary	Percentage of Participants With Adverse Events		5 years
Secondary	Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling		5 years