Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
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| ClinicalTrials.gov Identifier: NCT02369731 |
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Recruitment Status :
Recruiting
First Posted : February 24, 2015
Last Update Posted : February 14, 2017
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Sponsor:
PTC Therapeutics
Collaborators:
TREAT-NMD
Cooperative International Neuromuscular Research Group (CINRG)
Information provided by (Responsible Party):
PTC Therapeutics
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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 |
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 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 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Duchenne and Becker muscular dystrophy
MedlinePlus related topics:
Muscular Dystrophy
Intervention Details:
- Drug: Translarna
Other Names:
- Ataluren
- PTC124
Primary Outcome Measures :
- Incidence of adverse events [ Time Frame: 5 years ]
Secondary Outcome Measures :
- Changes in laboratory parameters [ Time Frame: 5 years ]
- Changes in blood pressure [ Time Frame: 5 years ]
- Prescriber and patient compliance with prescribing information according to the approved labelling [ Time Frame: 5 years ]
Other Outcome Measures:
- Patient health management measures [ Time Frame: 5 years ]
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | Child, Adult, Older Adult |
| 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02369731
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 |
| Responsible Party: | PTC Therapeutics |
| ClinicalTrials.gov Identifier: | NCT02369731 History of Changes |
| Other Study ID Numbers: |
PTC124-GD-025o-DMD |
| First Posted: | February 24, 2015 Key Record Dates |
| Last Update Posted: | February 14, 2017 |
| Last Verified: | February 2017 |
Additional relevant MeSH terms:
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Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |