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Trial record 1 of 27 for:    Idebenone
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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 : February 6, 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 available outside the clinical trial.   More info ...

Detailed Description:

The study will be 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). 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 2021
Estimated Study Completion Date : January 2022


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 Recruiting
Birmingham, Alabama, United States, 35233
Principal Investigator: Robert B. Troxler, MD         
United States, California
UC Davis Department of Physical Medicine and Rehabilitation Recruiting
Sacramento, California, United States, 95817
Principal Investigator: Craig McDonald, MD         
United States, Minnesota
Gillette Children's Specialty Healthcare Recruiting
Saint Paul, Minnesota, United States, 55101
Contact: Randel Richardson, 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
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage Recruiting
Paris, France, 75571
Principal Investigator: Teresa Gidaro, MD         
Pédiatrie Neurologie et infectiologie Pôle enfants Recruiting
Toulouse, France, 31059
Principal Investigator: Claude Cancès, MD         
Italy
Fondazione IRCCS Eugenio Medea Recruiting
Bosisio Parini, Italy, 23842
Contact: Maria Grazia Nadia D'Angelo, MD         
Principal Investigator: Maria Grazia Nadia D'Angelo, MD         
Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino" Recruiting
Messina, Italy, 98125
Principal Investigator: Giuseppe Vita, MD         
Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli Recruiting
Napoli, Italy, 80131
Principal Investigator: Luisa Politano, MD         
U.O.C. Neuropsichiatria Infantile Recruiting
Roma, Italy, 00168
Principal Investigator: Eugenio Mercuri, MD         
Spain
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     History of Changes
Other Study ID Numbers: SNT-III-012-E
First Posted: July 27, 2018    Key Record Dates
Last Update Posted: February 6, 2019
Last Verified: February 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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Idebenone
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
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Micronutrients
Nutrients
Growth Substances