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Trial record 1 of 1 for:    NCT03603288
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Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) (SIDEROS-E)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03603288
Recruitment Status : Recruiting
First Posted : July 27, 2018
Last Update Posted : October 14, 2019
Sponsor:
Information provided by (Responsible Party):
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.

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 ...

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 266 participants
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




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.



Information from the National Library of Medicine

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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
Criteria

Inclusion Criteria:

  1. Completion of the SIDEROS study at Visit 8/ Week 78
  2. Signed and dated Informed Consent Form for SIDEROS-E

Exclusion Criteria:

  1. Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8)
  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

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): NCT03603288


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

Locations
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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         
Sponsors and Collaborators
Santhera Pharmaceuticals

Additional Information:
Publications:
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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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Santhera Pharmaceuticals:
respiratory function in DMD
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
Ubiquinone
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Micronutrients
Nutrients
Growth Substances