Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) (SIDEROS-E)

• ClinicalTrials.gov Identifier: NCT03603288
• Recruitment Status: Recruiting
• First Posted: July 27, 2018
• Last Update Posted: October 14, 2019
• Sponsor: Santhera Pharmaceuticals
• Information provided by (Responsible Party): Santhera Pharmaceuticals

Study Description

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.

Study Design

• 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

Arms and Interventions

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

Outcome Measures

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.

Eligibility Criteria

• 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

Contacts and Locations

Contacts

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

Locations

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

More Information

Additional Information:

SIDEROS study website  SIDEROS study clinicaltrials.gov entry 

Publications:

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.

• Responsible Party: Santhera Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT03603288
• Other Study ID Numbers: SNT-III-012-E
• First Posted: July 27, 2018
• 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

Keywords provided by Santhera Pharmaceuticals:

respiratory function in DMD

Additional relevant MeSH terms:

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