Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
NCT ID: NCT03603288
Last Updated: 2021-12-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE3
161 participants
INTERVENTIONAL
2018-07-04
2020-11-25
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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idebenone 150 mg film-coated tablets
900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
idebenone 150 mg film-coated tablets
900 mg idebenone/day
Interventions
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idebenone 150 mg film-coated tablets
900 mg idebenone/day
Eligibility Criteria
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Inclusion Criteria
2. Signed and dated Informed Consent Form for SIDEROS-E
Exclusion Criteria
2. 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
3. Use of any investigational drug other than the study medication
11 Years
MALE
No
Sponsors
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Santhera Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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University of Alabama - Birmingham, Child Health Research
Birmingham, Alabama, United States
Banner University of Arizona Medical Center
Tucson, Arizona, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
UC Davis Department of Physical Medicine and Rehabilitation
Sacramento, California, United States
Center for Integrative Rare Disease Research, Rare Disease Research, LLC
Atlanta, Georgia, United States
University of Iowa, Department of Pediatrics
Iowa City, Iowa, United States
Johns Hopkins University
Baltimore, Maryland, United States
Children's Hospital Boston, Harvard Medical School, Department of Neurology
Boston, Massachusetts, United States
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
Charlotte, North Carolina, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
MetroHealth Medical Center
Cleveland, Ohio, United States
Children's Hospital of Philadelphia, Division of Pulmonology
Philadelphia, Pennsylvania, United States
Gottfried von Preyer'sches Kinderspital
Vienna, , Austria
University Hospital Leuven
Leuven, , Belgium
CHR Citadelle
Liège, , Belgium
Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
Lille, , France
CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie
Montpellier, , France
Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares
Nantes, , France
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage
Paris, , France
Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants
Toulouse, , France
University Medical Center Hamburg - Eppendorf, Department of Paediatrics
Hamburg, , Germany
Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München
München, , Germany
Fondazione IRCCS Eugenio Medea
Bosisio Parini, , Italy
U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini
Genova, , Italy
Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino"
Messina, , Italy
Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
Milan, , Italy
Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli
Napoli, , Italy
Reparto Di Neurologia dell'Osperdale Di Padova
Padua, , Italy
Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
Pavia, , 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
Countries
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References
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Buyse GM, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, McDonald CM, Rummey C, Meier T; DELOS Study Group. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial. Lancet. 2015 May 2;385(9979):1748-1757. doi: 10.1016/S0140-6736(15)60025-3. Epub 2015 Apr 20.
McDonald CM, Meier T, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Spagnolo P, Buyse GM; DELOS Study Group. Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2016 Aug;26(8):473-80. doi: 10.1016/j.nmd.2016.05.008. Epub 2016 May 12.
Buyse GM, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Mayer OH, Spagnolo P, Meier T, McDonald CM; DELOS Study Group. Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy. Pediatr Pulmonol. 2017 Apr;52(4):508-515. doi: 10.1002/ppul.23547. Epub 2016 Aug 29.
Mayer OH, Leinonen M, Rummey C, Meier T, Buyse GM; DELOS Study Group. Efficacy of Idebenone to Preserve Respiratory Function above Clinically Meaningful Thresholds for Forced Vital Capacity (FVC) in Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2017;4(3):189-198. doi: 10.3233/JND-170245.
Related Links
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SIDEROS study clinicaltrials.gov entry
Other Identifiers
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SNT-III-012-E
Identifier Type: -
Identifier Source: org_study_id