Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) (SIDEROS-E)
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ClinicalTrials.gov Identifier: NCT03603288 |
Recruitment Status :
Recruiting
First Posted : July 27, 2018
Last Update Posted : October 14, 2019
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: idebenone 150 mg film-coated tablets | Phase 3 |
Expanded Access : An investigational treatment associated with this study is temporarily not available outside the clinical trial. More info ...
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.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 266 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | Open-Label Extension Study |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Actual Study Start Date : | July 4, 2018 |
Estimated Primary Completion Date : | December 2023 |
Estimated Study Completion Date : | January 2024 |

Arm | Intervention/treatment |
---|---|
Experimental: idebenone 150 mg film-coated tablets
900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
|
Drug: idebenone 150 mg film-coated tablets
900 mg idebenone/day |
- Incidence and severity of adverse events, as per ICH Topic E2A [ Time Frame: From baseline until visit 4 (week 78) ]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 [ Time Frame: 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 premature discontinuations of study treatment due to adverse events. [ Time Frame: 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 safety laboratory parameters. [ Time Frame: 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 safety laboratory parameters. [ Time Frame: 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 vital signs. [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: From baseline until visit 4 (week 78) ]To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
- Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p). [ Time Frame: 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 Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) [ Time Frame: 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) [ Time Frame: 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.

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Ages Eligible for Study: | 11 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Completion of the SIDEROS study at Visit 8/ Week 78
- Signed and dated Informed Consent Form for SIDEROS-E
Exclusion Criteria:
- 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

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): NCT03603288
Contact: Jodi Wolff | +1 (520) 373-0069 | sideros@santhera.com |

United States, Alabama | |
University of Alabama - Birmingham, Child Health Research | Recruiting |
Birmingham, Alabama, United States, 35233 | |
Principal Investigator: Bradley Troxler, MD | |
United States, Arizona | |
Banner University of Arizona Medical Center | Recruiting |
Tucson, Arizona, United States, 85724 | |
Principal Investigator: Cori Daines, MD | |
United States, California | |
Children's Hospital of Los Angeles | Recruiting |
Los Angeles, California, United States, 90027 | |
Principal Investigator: Leigh Ramos-Platt, MD | |
UC Davis Department of Physical Medicine and Rehabilitation | Recruiting |
Sacramento, California, United States, 95817 | |
Principal Investigator: Craig McDonald, MD | |
United States, Georgia | |
Center for Integrative Rare Disease Research, Rare Disease Research, LLC | Recruiting |
Atlanta, Georgia, United States, 30318 | |
Principal Investigator: Han Phan, MD | |
United States, Iowa | |
University of Iowa, Department of Pediatrics | Recruiting |
Iowa City, Iowa, United States, 52242 | |
Principal Investigator: Kathy Matthews, MD | |
United States, Maryland | |
Johns Hopkins University | Recruiting |
Baltimore, Maryland, United States, 21287 | |
Principal Investigator: Thomas Crawford, MD | |
United States, Massachusetts | |
Children's Hospital Boston, Harvard Medical School, Department of Neurology | Recruiting |
Boston, Massachusetts, United States, 02115 | |
Principal Investigator: Basil Darras, MD | |
United States, Minnesota | |
Gillette Children's Specialty Healthcare | Recruiting |
Saint Paul, Minnesota, United States, 55101 | |
Principal Investigator: Randal Richardson, MD | |
United States, North Carolina | |
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System | Recruiting |
Charlotte, North Carolina, United States, 28207 | |
Contact: Johnny Jones, MS 704-446-1349 Johnny.Jones@carolinashealthcare.org | |
Contact: Lisa Ranzinger 704-446-0803 Lisa.Ranzinger@carolinashealthcare.org | |
Principal Investigator: Benjamin Brooks, MD | |
United States, Ohio | |
Cincinnati Children's Hospital | Recruiting |
Cincinnati, Ohio, United States, 45229 | |
Principal Investigator: Cuixia Tian, MD | |
MetroHealth Medical Center | Recruiting |
Cleveland, Ohio, United States, 44109-1988 | |
Contact: Alesia Cocklin, RN 216-778-2057 acocklin@metrohealth.org | |
Principal Investigator: Andre Prochoroff, MD | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia, Division of Pulmonology | Recruiting |
Philadelphia, Pennsylvania, United States, 19104-1771 | |
Principal Investigator: Oscar Henry Mayer, MD | |
Austria | |
Gottfried von Preyer'sches Kinderspital | Recruiting |
Vienna, Austria, 1100 | |
Principal Investigator: Bernert Guenther, MD | |
Belgium | |
University Hospital Leuven | Recruiting |
Leuven, Belgium, 3000 | |
Contact: Gunnar Buyse, MD | |
Principal Investigator: Gunnar Buyse, MD | |
CHR Citadelle | Recruiting |
Liège, Belgium, 4000 | |
Principal Investigator: Laurent Servais, MD | |
France | |
Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre | Recruiting |
Lille, France, 59037 | |
Principal Investigator: Jean-Marie Cuisset, MD | |
CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie | Recruiting |
Montpellier, France, 34295 | |
Principal Investigator: Ulkrike Walther-Louvier, MD | |
Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares | Recruiting |
Nantes, France, 44093 | |
Principal Investigator: Yan Pereon, MD | |
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage | Recruiting |
Paris, France, 75571 | |
Principal Investigator: Odile Boespflug-Tanguy, MD | |
Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants | Recruiting |
Toulouse, France, 31059 | |
Principal Investigator: Claude Cancès, MD | |
Germany | |
University Medical Center Hamburg - Eppendorf, Department of Paediatrics | Recruiting |
Hamburg, Germany, 20246 | |
Principal Investigator: Jessika Johannsen, MD | |
Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München | Recruiting |
München, Germany, 80337 | |
Principal Investigator: Wolfgang Muller-Felber, MD | |
Italy | |
Fondazione IRCCS Eugenio Medea | Recruiting |
Bosisio Parini, Italy, 23842 | |
Principal Investigator: Annamaria Russo, MD | |
U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini | Recruiting |
Genova, Italy, 16147 | |
Principal Investigator: Claudio Bruno, MD | |
Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino" | Recruiting |
Messina, Italy, 98125 | |
Principal Investigator: Giuseppe Vita, MD | |
Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital | Recruiting |
Milano, Italy, 20162 | |
Principal Investigator: Valeria Sansone, MD | |
Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli | Recruiting |
Napoli, Italy, 80131 | |
Principal Investigator: Luisa Politano, MD | |
Reparto Di Neurologia dell'Osperdale Di Padova | Recruiting |
Padova, Italy, 35122 | |
Principal Investigator: Elena Pegoraro, MD | |
Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia | Recruiting |
Pavia, Italy, 27100 | |
Principal Investigator: Angela Berardinelli, MD | |
U.O.C. Neuropsichiatria Infantile | Recruiting |
Roma, Italy, 00168 | |
Principal Investigator: Eugenio Mercuri, MD | |
Spain | |
Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia | Recruiting |
Barcelona, Spain, 08950 | |
Principal Investigator: Andres Nascimento, MD | |
Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D | Recruiting |
Valencia, Spain, 46026 | |
Principal Investigator: Juan Jesus Vilchez, MD | |
Switzerland | |
Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB) | Recruiting |
Basel, Switzerland, 4301 | |
Principal Investigator: Andrea Klein, MD | |
United Kingdom | |
Leeds Teaching Hospital NHS Trust | Recruiting |
Leeds, United Kingdom, LS1 3EX | |
Principal Investigator: Anne-Marie Childs, MD | |
UCL, National Hospital for Neurology and Neurosurgery | Recruiting |
London, United Kingdom, WC1 3BG | |
Principal Investigator: Rosaline Quinlivan, MD | |
Great Ormond Street Hospital for Children | Recruiting |
London, United Kingdom, WC1N 3JH | |
Principal Investigator: Pinki Munot, MD | |
Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary | Recruiting |
Newcastle Upon Tyne, United Kingdom, NE1 4LP | |
Principal Investigator: Michela Guglieri, MD | |
Robert Jones and Agnes Hunt Orthopaedic Hospital | Recruiting |
Oswestry, United Kingdom, SY10 7AG | |
Contact: Tracey Willis, MD | |
Principal Investigator: Tracey Willis, MD |
Publications:
Responsible Party: | Santhera Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03603288 |
Other Study ID Numbers: |
SNT-III-012-E |
First Posted: | July 27, 2018 Key Record Dates |
Last Update Posted: | October 14, 2019 |
Last Verified: | October 2019 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
respiratory function in DMD |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases |
Genetic Diseases, Inborn Genetic Diseases, X-Linked Idebenone Antioxidants Molecular Mechanisms of Pharmacological Action Protective Agents Physiological Effects of Drugs |