A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study
Who is this study for? Child to adult male patients with Duchenne Muscular Dystrophy
What treatments are being studied? Idebenone
Status: Terminated
Location: See all (39) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 11
Healthy Volunteers: f
View:
• Completion of the SIDEROS study at Visit 8/ Week 78
• Signed and dated Informed Consent Form for SIDEROS-E
Locations
United States
Alabama
University of Alabama - Birmingham, Child Health Research
Birmingham
Arizona
Banner University of Arizona Medical Center
Tucson
California
Children's Hospital of Los Angeles
Los Angeles
UC Davis Department of Physical Medicine and Rehabilitation
Sacramento
Georgia
Center for Integrative Rare Disease Research, Rare Disease Research, LLC
Atlanta
Iowa
University of Iowa, Department of Pediatrics
Iowa City
Massachusetts
Children's Hospital Boston, Harvard Medical School, Department of Neurology
Boston
Maryland
Johns Hopkins University
Baltimore
Minnesota
Gillette Children's Specialty Healthcare
Saint Paul
North Carolina
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
Charlotte
Ohio
Cincinnati Children's Hospital
Cincinnati
MetroHealth Medical Center
Cleveland
Pennsylvania
Children's Hospital of Philadelphia, Division of Pulmonology
Philadelphia
Other Locations
Austria
Gottfried von Preyer'sches Kinderspital
Vienna
Belgium
University Hospital Leuven
Leuven
CHR Citadelle
Liège
France
Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
Lille
CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie
Montpellier
Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares
Nantes
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage
Paris
Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants
Toulouse
Germany
University Medical Center Hamburg - Eppendorf, Department of Paediatrics
Hamburg
Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München
München
Italy
Fondazione IRCCS Eugenio Medea
Bosisio Parini
U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini
Genova
Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico G. Martino
Messina
Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
Milano
Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli
Napoli
Reparto Di Neurologia dell'Osperdale Di Padova
Padova
Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS C. Mondino di Pavia
Pavia
U.O.C. Neuropsichiatria Infantile
Roma
Spain
Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia
Barcelona
Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D
Valencia
Switzerland
Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
Basel
United Kingdom
Leeds Teaching Hospital NHS Trust
Leeds
Great Ormond Street Hospital for Children
London
UCL, National Hospital for Neurology and Neurosurgery
London
Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary
Newcastle Upon Tyne
Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry
Time Frame
Start Date: 2018-07-04
Completion Date: 2020-11-25
Participants
Target number of participants: 161
Treatments
Experimental: idebenone 150 mg film-coated tablets
900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
Authors
Bradley Troxler, Leigh Ramos-Platt, Benjamin Brooks, Robert B. Troxler, Cuixia Tian, Randal Richardson, Andre Prochoroff, Basil Darras, Oscar Henry Mayer, Cori Daines, Carla Grosmann, Han Phan, Teresa Gidaro, Jessika Johannsen, Laurent Servais, Rosaline Quinlivan, Wolfgang Muller-Felber, Odile Boespflug-Tanguy, Claude Cancès, Claudio Bruno, Craig McDonald, Giuseppe Vita, Jean-Marie Cuisset, Gunnar Buyse, Andrea Klein, Tracey Willis, Angela Berardinelli, Annamaria Russo, Valeria Sansone, Elena Pegoraro, Andres Nascimento, Juan Jesus Vilchez, Luisa Politano, Michela Guglieri, Anne-Marie Childs, Pinki Munot, Eugenio Mercuri
Related Therapeutic Areas
Sponsors
Leads: Santhera Pharmaceuticals