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

Duchenne Muscular Dystrophy Clinical Trial

— SIDEROS-E  

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03603288
• Other study ID #: SNT-III-012-E
• Status: Terminated
• Phase: Phase 3
• Start date: July 4, 2018
• Est. completion date: November 25, 2020

Study information

• Verified date: November 2021
• Source: Santhera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 161
• Est. completion date: November 25, 2020
• Est. primary completion date: November 25, 2020
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 11 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• idebenone 150 mg film-coated tablets
900 mg idebenone/day

Locations

Country	Name	City	State
Austria	Gottfried von Preyer'sches Kinderspital	Vienna
Belgium	University Hospital Leuven	Leuven
Belgium	CHR Citadelle	Liège
France	Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre	Lille
France	CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie	Montpellier
France	Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares	Nantes
France	I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage	Paris
France	Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants	Toulouse
Germany	University Medical Center Hamburg - Eppendorf, Department of Paediatrics	Hamburg
Germany	Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München	München
Italy	Fondazione IRCCS Eugenio Medea	Bosisio Parini
Italy	U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini	Genova
Italy	Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino"	Messina
Italy	Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital	Milano
Italy	Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli	Napoli
Italy	Reparto Di Neurologia dell'Osperdale Di Padova	Padova
Italy	Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia	Pavia
Italy	U.O.C. Neuropsichiatria Infantile	Roma
Spain	Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia	Barcelona
Spain	Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D	Valencia
Switzerland	Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)	Basel
United Kingdom	Leeds Teaching Hospital NHS Trust	Leeds
United Kingdom	Great Ormond Street Hospital for Children	London
United Kingdom	UCL, National Hospital for Neurology and Neurosurgery	London
United Kingdom	Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary	Newcastle Upon Tyne
United Kingdom	Robert Jones and Agnes Hunt Orthopaedic Hospital	Oswestry
United States	Center for Integrative Rare Disease Research, Rare Disease Research, LLC	Atlanta	Georgia
United States	Johns Hopkins University	Baltimore	Maryland
United States	University of Alabama - Birmingham, Child Health Research	Birmingham	Alabama
United States	Children's Hospital Boston, Harvard Medical School, Department of Neurology	Boston	Massachusetts
United States	Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System	Charlotte	North Carolina
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	MetroHealth Medical Center	Cleveland	Ohio
United States	University of Iowa, Department of Pediatrics	Iowa City	Iowa
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	Children's Hospital of Philadelphia, Division of Pulmonology	Philadelphia	Pennsylvania
United States	UC Davis Department of Physical Medicine and Rehabilitation	Sacramento	California
United States	Gillette Children's Specialty Healthcare	Saint Paul	Minnesota
United States	Banner University of Arizona Medical Center	Tucson	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Countries where clinical trial is conducted

United States,  Austria,  Belgium,  France,  Germany,  Italy,  Spain,  Switzerland,  United Kingdom, 

References & Publications (4)

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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
Primary	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)
Primary	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)
Primary	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
Primary	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)
Primary	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
Primary	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	Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p).	To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.	From baseline until visit 4 (week 78)
Secondary	Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)	To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.	From baseline until visit 4 (week 78)
Secondary	Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p)	To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.	From baseline until visit 4 (week 78)

External Links

•   SIDEROS study clinicaltrials.gov entry
•   SIDEROS study website