Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Registration Number
- NCT03603288
- Lead Sponsor
- Santhera Pharmaceuticals
- Brief 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.
- Detailed Description
The study is an open-label, single-group, multi-center extension study in patients with DMD receiving glucocorticoid steroids who participated in the SIDEROS study and who meet all the inclusion criteria and none of the exclusion criteria for this extension study.
The study consists of 4 study visits scheduled every 6 months (Visit 1/Baseline, Visit 2/Week 26, Visit 3/ Week 52 and Visit 4/ Week 78), and a follow-up visit 4 weeks after treatment discontinuation. Visit 8/Week 78 in SIDEROS study is also SIDEROS-E Visit 1/Baseline.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 161
- Completion of the SIDEROS study at Visit 8/ Week 78
- Signed and dated Informed Consent Form for SIDEROS-E
- Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8)
- Safety, tolerability or other issues arising during the course of the SIDEROS study which in the opinion of the Investigator may put the patient at significant risk or may interfere significantly with the patient's participation in the SIDEROS-E study
- Use of any investigational drug other than the study medication
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description idebenone 150 mg film-coated tablets idebenone 150 mg film-coated tablets 900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
- Primary Outcome Measures
Name Time Method Number of patients with premature discontinuations of study treatment due to adverse events. From baseline until visit 4 (week 78) To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal vital signs. 4 weeks after discontinuation of treatment To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Incidence and severity of adverse events, as per ICH Topic E2A 4 weeks after discontinuation of treatment To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal safety laboratory parameters. 4 weeks after discontinuation of treatment To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal ECG. From baseline until visit 4 (week 78) To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
- Secondary Outcome Measures
Name Time Method Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) From baseline until visit 4 (week 78) To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p) From baseline until visit 4 (week 78) To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p). From baseline until visit 4 (week 78) To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Trial Locations
- Locations (39)
Gillette Children's Specialty Healthcare
🇺🇸Saint Paul, Minnesota, United States
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
University of Alabama - Birmingham, Child Health Research
🇺🇸Birmingham, Alabama, United States
Children's Hospital Boston, Harvard Medical School, Department of Neurology
🇺🇸Boston, Massachusetts, United States
Center for Integrative Rare Disease Research, Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
UC Davis Department of Physical Medicine and Rehabilitation
🇺🇸Sacramento, California, United States
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
University of Iowa, Department of Pediatrics
🇺🇸Iowa City, Iowa, United States
MetroHealth Medical Center
🇺🇸Cleveland, Ohio, United States
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
🇺🇸Charlotte, North Carolina, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares
🇫🇷Nantes, France
Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants
🇫🇷Toulouse, France
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage
🇫🇷Paris, France
University Medical Center Hamburg - Eppendorf, Department of Paediatrics
🇩🇪Hamburg, Germany
U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini
🇮🇹Genova, Italy
Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino"
🇮🇹Messina, Italy
Fondazione IRCCS Eugenio Medea
🇮🇹Bosisio Parini, Italy
Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
🇮🇹Milano, Italy
Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli
🇮🇹Napoli, Italy
Reparto Di Neurologia dell'Osperdale Di Padova
🇮🇹Padova, Italy
U.O.C. Neuropsichiatria Infantile
🇮🇹Roma, Italy
Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia
🇪🇸Barcelona, Spain
Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D
🇪🇸Valencia, Spain
Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
🇨🇭Basel, Switzerland
Leeds Teaching Hospital NHS Trust
🇬🇧Leeds, United Kingdom
UCL, National Hospital for Neurology and Neurosurgery
🇬🇧London, United Kingdom
Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom
Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary
🇬🇧Newcastle Upon Tyne, United Kingdom
Robert Jones and Agnes Hunt Orthopaedic Hospital
🇬🇧Oswestry, United Kingdom
CHR Citadelle
🇧🇪Liège, Belgium
Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
🇮🇹Pavia, Italy
Gottfried von Preyer'sches Kinderspital
🇦🇹Vienna, Austria
University Hospital Leuven
🇧🇪Leuven, Belgium
Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
🇫🇷Lille, France
Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München
🇩🇪München, Germany
CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie
🇫🇷Montpellier, France
Children's Hospital of Philadelphia, Division of Pulmonology
🇺🇸Philadelphia, Pennsylvania, United States