Clinical trial NCT03603288: Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Duchenne Muscular Dystrophy
Keywords: respiratory function in DMD
Lead sponsor: Santhera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2018-07-27
Enrollment: 161
Sex: MALE
Minimum age (years): 11
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-Label Extension Study
Primary purpose: TREATMENT

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.

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.

Eligibility 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

Primary outcomes:
measure: Incidence and severity of adverse events, as per ICH Topic E2A
timeFrame: From baseline until visit 4 (week 78)
measure: Incidence and severity of adverse events, as per ICH Topic E2A
timeFrame: 4 weeks after discontinuation of treatment
measure: Number of patients with premature discontinuations of study treatment due to adverse events.
timeFrame: From baseline until visit 4 (week 78)
measure: Number of patients with abnormal safety laboratory parameters.
timeFrame: From baseline until visit 4 (week 78)
measure: Number of patients with abnormal safety laboratory parameters.
timeFrame: 4 weeks after discontinuation of treatment
measure: Number of patients with abnormal vital signs.
timeFrame: From baseline until visit 4 (week 78)
measure: Number of patients with abnormal vital signs.
timeFrame: 4 weeks after discontinuation of treatment
measure: Number of patients with abnormal ECG.
timeFrame: From baseline until visit 4 (week 78)
Secondary outcomes:
measure: Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p).
timeFrame: From baseline until visit 4 (week 78)
measure: Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)
timeFrame: From baseline until visit 4 (week 78)
measure: Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p)
timeFrame: From baseline until visit 4 (week 78)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama - Birmingham, Child Health Research
state: Alabama
city: Tucson
country: United States
facility: Banner University of Arizona Medical Center
state: Arizona
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Sacramento
country: United States
facility: UC Davis Department of Physical Medicine and Rehabilitation
state: California
city: Atlanta
country: United States
facility: Center for Integrative Rare Disease Research, Rare Disease Research, LLC
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa, Department of Pediatrics
state: Iowa
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Children's Hospital Boston, Harvard Medical School, Department of Neurology
state: Massachusetts
city: Saint Paul
country: United States
facility: Gillette Children's Specialty Healthcare
state: Minnesota
city: Charlotte
country: United States
facility: Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Cleveland
country: United States
facility: MetroHealth Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia, Division of Pulmonology
state: Pennsylvania
city: Vienna
country: Austria
facility: Gottfried von Preyer'sches Kinderspital
city: Leuven
country: Belgium
facility: University Hospital Leuven
city: Liège
country: Belgium
facility: CHR Citadelle
city: Lille
country: France
facility: Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
city: Montpellier
country: France
facility: CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie
city: Nantes
country: France
facility: Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares
city: Paris
country: France
facility: I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage
city: Toulouse
country: France
facility: Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants
city: Hamburg
country: Germany
facility: University Medical Center Hamburg - Eppendorf, Department of Paediatrics
city: München
country: Germany
facility: Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München
city: Bosisio Parini
country: Italy
facility: Fondazione IRCCS Eugenio Medea
city: Genova
country: Italy
facility: U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini
city: Messina
country: Italy
facility: Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino"
city: Milano
country: Italy
facility: Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
city: Napoli
country: Italy
facility: Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli
city: Padova
country: Italy
facility: Reparto Di Neurologia dell'Osperdale Di Padova
city: Pavia
country: Italy
facility: Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
city: Roma
country: Italy
facility: U.O.C. Neuropsichiatria Infantile
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia
city: Valencia
country: Spain
facility: Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D
city: Basel
country: Switzerland
facility: Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospital NHS Trust
city: London
country: United Kingdom
facility: UCL, National Hospital for Neurology and Neurosurgery
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: Newcastle Upon Tyne
country: United Kingdom
facility: Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary
city: Oswestry
country: United Kingdom
facility: Robert Jones and Agnes Hunt Orthopaedic Hospital
Study officials:
References:
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.
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.
citation: 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.
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.