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

NCT03603288 | Terminated Phase 3 DMC FDA Drug

• 1 other identifier: SNT-III-012-E
• Study Type: interventional
• Target: 161
• Locations: 9 countries
• Sites: 39

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

respiratory function in DMD

Outcome Measures

Primary Outcomes (8)

• Incidence and severity of adverse events, as per ICH Topic E2A

  To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

  From baseline until visit 4 (week 78)

• Incidence and severity of adverse events, as per ICH Topic E2A

  To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

  4 weeks after discontinuation of treatment

• Number of patients with premature discontinuations of study treatment due to adverse events.

  To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

  From baseline until visit 4 (week 78)

• Number of patients with abnormal safety laboratory parameters.

  To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

  From baseline until visit 4 (week 78)

• Number of patients with abnormal safety laboratory parameters.

  To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

  4 weeks after discontinuation of treatment

• Number of patients with abnormal vital signs.

  To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

  From baseline until visit 4 (week 78)

• Number of patients with abnormal vital signs.

  To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

  4 weeks after discontinuation of treatment

• Number of patients with abnormal ECG.

  To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

  From baseline until visit 4 (week 78)

Secondary Outcomes (3)

• Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p).

  From baseline until visit 4 (week 78)

• Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)

  From baseline until visit 4 (week 78)

• Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p)

  From baseline until visit 4 (week 78)

Study Arms (1)

idebenone 150 mg film-coated tablets

EXPERIMENTAL

900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)

Drug: idebenone 150 mg film-coated tablets

Interventions

idebenone 150 mg film-coated tablets DRUG

900 mg idebenone/day

idebenone 150 mg film-coated tablets

Eligibility Criteria

• Age: 11 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (39)

University of Alabama - Birmingham, Child Health Research

Birmingham, Alabama, 35233, United States

Location

Banner University of Arizona Medical Center

Tucson, Arizona, 85724, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

UC Davis Department of Physical Medicine and Rehabilitation

Sacramento, California, 95817, United States

Location

Center for Integrative Rare Disease Research, Rare Disease Research, LLC

Atlanta, Georgia, 30318, United States

Location

University of Iowa, Department of Pediatrics

Iowa City, Iowa, 52242, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

Children's Hospital Boston, Harvard Medical School, Department of Neurology

Boston, Massachusetts, 02115, United States

Location

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States

Location

Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System

Charlotte, North Carolina, 28207, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

MetroHealth Medical Center

Cleveland, Ohio, 44109-1988, United States

Location

Children's Hospital of Philadelphia, Division of Pulmonology

Philadelphia, Pennsylvania, 19104-1771, United States

Location

Gottfried von Preyer'sches Kinderspital

Vienna, 1100, Austria

Location

University Hospital Leuven

Leuven, 3000, Belgium

Location

CHR Citadelle

Liège, 4000, Belgium

Location

Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre

Lille, 59037, France

Location

CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie

Montpellier, 34295, France

Location

Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares

Nantes, 44093, France

Location

I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage

Paris, 75571, France

Location

Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants

Toulouse, 31059, France

Location

University Medical Center Hamburg - Eppendorf, Department of Paediatrics

Hamburg, 20246, Germany

Location

Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München

München, 80337, Germany

Location

Fondazione IRCCS Eugenio Medea

Bosisio Parini, 23842, Italy

Location

U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini

Genova, 16147, Italy

Location

Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino"

Messina, 98125, Italy

Location

Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital

Milan, 20162, Italy

Location

Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli

Napoli, 80131, Italy

Location

Reparto Di Neurologia dell'Osperdale Di Padova

Padua, 35122, Italy

Location

Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia

Pavia, 27100, Italy

Location

U.O.C. Neuropsichiatria Infantile

Roma, 00168, Italy

Location

Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia

Barcelona, 08950, Spain

Location

Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D

Valencia, 46026, Spain

Location

Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)

Basel, 4301, Switzerland

Location

Leeds Teaching Hospital NHS Trust

Leeds, LS1 3EX, United Kingdom

Location

UCL, National Hospital for Neurology and Neurosurgery

London, WC1 3BG, United Kingdom

Location

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

Location

Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary

Newcastle upon Tyne, NE1 4LP, United Kingdom

Location

Robert Jones and Agnes Hunt Orthopaedic Hospital

Oswestry, SY10 7AG, United Kingdom

Location

Related Publications (4)

• 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.

  PMID: 25907158 BACKGROUND

• 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.

  PMID: 27238057 BACKGROUND

• 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.

  PMID: 27571420 BACKGROUND

• 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.

  PMID: 28869486 BACKGROUND

Related Links

• SIDEROS study clinicaltrials.gov entry

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

idebenone

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Model Details: Open-Label Extension Study

Study Record Dates

• First Submitted: May 31, 2018
• First Posted: July 27, 2018
• Study Start: July 4, 2018
• Primary Completion: November 25, 2020
• Study Completion: November 25, 2020
• Last Updated: December 3, 2021

Record last verified: 2021-11

Locations

BE (2); DE (2); IT (8); ES (2); CH (1); US (13); AU (1); GB (5); FR (5)