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

Study Description

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

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.

Condition or disease	Intervention/treatment
Muscular Dystrophy, Duchenne	Drug: Translarna

Detailed Description:

This is a multicenter, observational study of patients receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 200 patients across ~50 care centers in Europe and other regions over a period of ~ 2 years. The study population will include patients who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named patient early access program) and who provide consent. Patients 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 Design

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Study Type :	Observational [Patient Registry]
Estimated Enrollment :	200 participants
Observational Model:	Cohort
Time Perspective:	Prospective
Target Follow-Up Duration:	5 Years
Official Title:	Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care
Study Start Date :	April 2015
Estimated Primary Completion Date :	April 2022
Estimated Study Completion Date :	April 2022

Groups and Cohorts

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Intervention Details:

Drug: Translarna
Other Names:
• Ataluren
• PTC124

Outcome Measures

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Primary Outcome Measures :

1. Incidence of adverse events [ Time Frame: 5 years ]

Secondary Outcome Measures :

1. Changes in laboratory parameters [ Time Frame: 5 years ]
2. Changes in blood pressure [ Time Frame: 5 years ]
3. Prescriber and patient compliance with prescribing information according to the approved labelling [ Time Frame: 5 years ]

Other Outcome Measures:

1. Patient health management measures [ Time Frame: 5 years ]

Eligibility Criteria

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Ages Eligible for Study:	Child, Adult, Senior
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No
Sampling Method:	Probability Sample

Study Population

The target study population will include patients for whom the decision to prescribe Translarna (outside of a clinical trial) has already been made, including patients in named patient early access programs. Patients 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 patient or through authorisation by a legal guardian), will be invited to enroll into the study and will be followed according to the protocol.

Criteria

Inclusion Criteria:

• Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named patient early access program)
• Willing to provide written informed consent to allow the study data collection procedures (either by the patient or through authorisation by a legal guardian)

Exclusion Criteria:

• Patients 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

Contacts and Locations

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Contacts

Contact: Abdallah Delage		adelage@ptcbio.com

Locations

Austria
Krankenhaus der Barmherzigen Schwestern Linz	Recruiting
Linz, Austria, 4010
Principal Investigator: Veronika Pilshofer, Dr
Preyer Children Hospital	Recruiting
Wien, Austria, 1100
Principal Investigator: Guenther Bernert, Pr
France
CHU Brest, hôpital Morvan	Recruiting
Best, France, 29609
Principal Investigator: Ropars Juliette, Dr
CHU Bordeaux Pelgrin	Recruiting
Bordeaux Cedex, France, 33076
Principal Investigator: Caroline Espil-Taris
L'Escale Hôpital Femme Mère Enfant- Hospices Civils de Lyon	Recruiting
Bron, France, 69500
Principal Investigator: Carole Vuillerot, Dr
CHU Clermont-Ferrand, hôpital Estaing	Recruiting
Clermont-Ferrand, France, 63003
Principal Investigator: Catherine Sarret, Dr
CHU de Martinique - Hôpital P. Zobda-Quitman	Recruiting
Fort De France Cedex, France, 97261
Principal Investigator: Rémi Bellance, Dr
CHRU de Lille	Recruiting
Lille Cedex, France, 59037
Principal Investigator: Jean-Marie Cuisset, Dr
CHU Marseille Hôpital de la Timone	Recruiting
Marseille Cedex 5, France, 13385
Principal Investigator: Brigitte Chabrol, Pr
CHU Nantes	Recruiting
Nantes, France, 44000
Principal Investigator: Yan Pereon, Pr
Hôpital Armand Trousseau	Recruiting
Paris, France, 75012
Principal Investigator: Gidaro Teresa, Dr
Hôpital Necker	Recruiting
Paris, France, 75015
Principal Investigator: Isabelle Desguerre, Pr
CHU Reims	Recruiting
Reims, France, 51100
Principal Investigator: Pascal Sabouraud, Dr
CHU Saint-Etienne MPR pédiatrique Hôpital Bellevue	Recruiting
Saint Etienne Cedex, France, 42055
Principal Investigator: Stéphane Chabrier, Dr
CHU de la Réunion - GHSR - GH Sud-Réunion	Recruiting
Saint Pierre, France, 97448
Principal Investigator: Renouil Michel, Dr
CHU Strasbourg	Recruiting
Strasbourg Cedex, France, 67098
Principal Investigator: Vincent Laugel, Pr
CHU Toulouse	Recruiting
Toulouse, France, 31059
Principal Investigator: Pascal Cintas, Dr
Hôpital d'enfants Gatien de Clocheville	Recruiting
Tours Cedex 9, France, 37044
Principal Investigator: Lagrue Emmanuelle, Dr
CHU Nancy Hôpital de Brabois-Enfants Rue du Morvan	Recruiting
Vandoeuvre-les-Nancy, France, 54500
Principal Investigator: Emmanuel Raffo, Pr
Germany
Klinikum Bayreuth GmbH	Recruiting
Bayreuth, Germany, 95445
Principal Investigator: Almut Hirsch, Dr
Charité Universitätsmedizin Berlin	Recruiting
Berlin, Germany, 13353
Principal Investigator: Claudia Weiss, Dr
Universitätsklinikum Erlangen	Recruiting
Erlangen, Germany, 91054
Principal Investigator: Regina Trollmann, Pr
Universitaetsklinikum Essen (AoR)	Recruiting
Essen, Germany, 45147
Principal Investigator: Ulrike Schara, Pr
SPZ Frankfurt Mitte	Recruiting
Frankfurt am Main, Germany, 60316
Principal Investigator: Jurgen Seeger, Dr
Universitaetsklinikum Freiburg	Recruiting
Freiburg, Germany, 79106
Principal Investigator: Janbernd Kirschner, Pr
UKE, Universitätsklinikum Hamburg-Eppendorf	Recruiting
Hamburg, Germany, 20246
Principal Investigator: Jessica Johannsen, Dr
Israel
Hadassah Medical Center	Recruiting
Jerusalem, Israel, 91120
Principal Investigator: Talya Dor, Dr
Chaim Sheba Medical Center Ramat Gan, Neuromuscle clinic	Recruiting
Ramat Gan, Israel, 52621
Principal Investigator: Amir Dori, Dr
Sweden
Queen Silvia Children's Hospital	Recruiting
Goteborg, Sweden, 41685
Principal Investigator: Mar Tulinius, Pr

Sponsors and Collaborators

PTC Therapeutics

TREAT-NMD

Cooperative International Neuromuscular Research Group (CINRG)

Investigators

Study Director:	Robert Spiegel, MD	PTC Therapeutics

More Information

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	PTC Therapeutics
ClinicalTrials.gov Identifier:	NCT02369731 History of Changes
Other Study ID Numbers:	PTC124-GD-025o-DMD
First Posted:	February 24, 2015
Last Update Posted:	February 14, 2017
Last Verified:	February 2017

Additional relevant MeSH terms:

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases	Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked