Last updated on April 2018

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

Brief description of study

The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids

Detailed Study Description

The SIDEROS trial is a randomized, placebo controlled, parallel group study of the efficacy of idebenone in delaying the loss of respiratory function, whilst also monitoring safety and tolerability of idebenone in at least 266 DMD patients taking stable dose of concomitant glucocorticoid steroids.

The study treatment period will be 18 months/ 78 weeks and the idebenone dose will be 900 mg/day. Participants can use deflazacort or prednisolone and be on any dose regimen.

Since glucocorticoid steroids are widely used in ambulant boys from an early age until late into teenage and even adult years, this study will not take age and ambulatory status into account and will only exclude patients that need daytime ventilator assistance.

The schedule of assessments will include a Screening Visit and up to 9 protocol visits, including a Follow-up Visit.

A Screening Visit will take place a maximum of 6 weeks prior to the Baseline Visit (Visit 1, study day -1). Beginning at Baseline, the patient will receive study medication to be taken at home, and will undergo regular assessments in the clinic throughout the study period until Visit 8 at Week 78 at which time the study will be completed and medication discontinued.

All patients completing Visit 8/Week 78, and considered eligible by the Investigator will be able to participate in an open-label extension study (SIDEROS-E) and will continue to receive idebenone until the SIDEROS-E is terminated or Marketing Authorization is obtained for idebenone in DMD, whichever occurs first. The duration of the SIDEROS-E study will be defined in a separate protocol.

For all patients not participating in the extension study (SIDEROS-E), a Follow-up Visit (Visit 9/Follow-up Visit) will take place 4 weeks after end of Treatment at Visit 8/Week 78 or after premature discontinuation of study medication.

Each hospital visit will include efficacy assessments (respiratory function assessed by hospital-based spirometry, oxygen saturation, end-tidal CO2) and safety assessments (adverse events, concomitant medication, physical examination, vital signs, safety laboratory evaluations). In addition, respiratory function will be assessed weekly at home with a hand-held device in order to closely monitor respiratory function between hospital visits.

The study medication, all medical procedures and laboratory testing, and the visits to the study centre are free of charge. In addition the patients will receive a travel allowance to cover reasonable expenses to and from the study centre. Participants will not otherwise be compensated for this study.

Clinical Study Identifier: NCT02814019

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Bradley Troxler, MD

University of Alabama
Birmingham, AL United States
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James Woodward, MD

Phoenix Children's Hospital
Phoenix, AZ United States
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Cori Daines, MD

Banner University of Arizona Medical Center
Tucson, AZ United States
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Vikki Stefans, MD

Arkansas Children's Hospital
Little Rock, AR United States
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Perry Shieh, MD

David Geffen School of Medicine at UCLA
Los Angeles, CA United States
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Leigh Maria Ramos Platt

Childrens Hospital of Los Angeles
Los Angeles, CA United States
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Craig McDonald, MD

UC Davis Department of Physical Medicine and Rehabilitation
Sacramento, CA United States
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David Michelson

Loma Linda University Healthcare
San Bernardino, CA United States
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Marisia Couluris, MD

Shriners Hospitals for Children-Tampa
Tampa, FL United States
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Han Phan, MD

Rare Disease Research
Atlanta, GA United States
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Kathy Mathews, MD

University of Iowa
Iowa City, IA United States
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Jeff Statland, MD

University of Kansas
Fairway, KS United States
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Thomas Crawford, MD

Johns Hopkins University
Baltimore, MD United States
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Basil Darras, MD

Children's Hospital Boston
Boston, MA United States
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Randel Richardson

Gillette Children's Specialty Healthcare
Saint Paul, MN United States
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Benjamin Brooks, MD

Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
Charlotte, NC United States
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Cuixia Tian, MD

Cincinnati Children's Hospital
Cincinnati, OH United States
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Andre Prochoroff, MD

MetroHealth Medical Center
Cleveland, OH United States
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Oscar Mayer, MD

Children's Hospital of Philadelphia
Philadelphia, PA United States
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Tulio Bertorini, MD

Wesley Neurology Clinic
Memphis, TN United States
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Warren Marks, MD

Cook Children's Medical Center
Fort Worth, TX United States
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Russell Butterfield, MD

University of Utah
Salt Lake City, UT United States
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Bernert Guenther, MD

Gottfried von Preyer'sches Kinderspital
Wien, Austria
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Gunnar Buyse, MD

University Hospital Leuven
Leuven, Belgium
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Jean-Marie Cuisset, MD

Service de neurop diatrie P le P diatrie CHRU de Lille - H pital Jeanne de Flandre
Lille, France
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Ulrike Walther-Louvier, MD

CHRU de Montpellier - H pital Gui de Chauliac
Montpellier, France
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Yann Pereon

H pital H tel Dieu
Nantes, France
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Teresa Gidaro, MD

I-Motion - Plateforme d'essais cliniques p diatriques H pital Armand Trousseau b timent Lemariey porte 20
Paris, France
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Claude Cancès, MD

H pital des enfants
Toulouse, France
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Angela Kaindl

Universit tsmedizin Berlin Campus Virchow-Klinikum
Berlin, Germany
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Ulrike Schara

Universit tsklinikum Essen
Essen, Germany
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Janbernd Kirschner, MD

Universit tsklinik Freiburg Zentrum f r Kinderheilkunde und Jugendmedizin
Freiburg, Germany
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Jessika Johannsen

Universit tsklinikum Hamburg-Eppendorf, Klinik f r Kinder- und Jugendmedizin
Hamburg, Germany
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Wolfgang Müller-Felber, MD

Center for neuromuscular disorders
Munich, Germany
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Yoram Nevo, MD

Institute of Neurology at Schneider Children's Medical Center of Israel
Petah Tiqva, Israel
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Maria Grazia Nadia D'Angelo, MD

Fondazione IRCCS Eugenio Medea
Bosisio Parini, Italy
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Claudio Bruno, MD

Istituto Giannina Gaslini
Genova, Italy
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Giuseppe Vita, MD

Scientific Coordinator Nemo Sud Clinical Center
Messina, Italy
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Valeria Sansone, MD

Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
Milano, Italy
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Luisa Politano, MD

Servizio di Cardiomiologia e Genetica Medica, AOU Universit degli Studi della Campania "Luigi Vanvitelli"
Napoli, Italy
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Elena Pegoraro

Reparto Di Neurologia dell'Osperdale Di Padova
Padova, Italy
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Angela Berardinelli

Dipartimento di Clinica Neurologica e Psichiatrica dell'Et Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
Pavia, Italy
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Eugenio Mercuri, MD

U.O.C. Neuropsichiatria Infantile
Roma, Italy
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Chiara Straathof, MD

Leiden, Netherlands
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Imelda J M De Groot, ...

Radboud university medical centre
Nijmegen, Netherlands
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Andres Nascimento, MD

Hospital Sant Joan de D u Neuropediatra
Barcelona, Spain
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Francina Munell, MD

Hospital Universitari Vall D' Hebron
Barcelona, Spain
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Samuel Pascual

Hospital Universitario La Paz
Madrid, Spain
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Marcos Madruga

Hospital Universitario Virgen del Rocio
Sevilla, Spain
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Juan Jesus Vilchez, MD

Hospital La Fe de Valencia
Valencia, Spain
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Mar Tulinius, MD

Sahlgrenska University Hospital
Gothenburg, Sweden
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Andrea Klein, MD

Center for neuromuscular disorders, Universit ts-Kinderspital beider Basel (UKBB)
Basel, Switzerland
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Anne-Marie Childs

Leeds Teaching Hospital NHS Trust
Leeds, United Kingdom
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Rosaline Quinlivan

UCL, National Hospital for Neurology and Neurosurgery
London, United Kingdom
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Pinki Munot

Great Ormond Street Hospital for Children
London, United Kingdom
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Michela Guglieri, MD

John Walton Muscular Dystrophy Research Centre
Newcastle, United Kingdom
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Tracey Willis

Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry, United Kingdom
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University of Rochester Medical Center
Rochester, NY United States
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Laurent Servais, MD

Centre de R f rence Neuromusculaire, CHR Citadelle
Liège, Belgium
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Ivailo Tournev

Sofia Medical University
Sofia, Bulgaria
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Sebahattin Cirak, MD

Uniklinik K ln
Köln, Germany
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Declan O'Rourke

Children's University Hospital
Dublin, Ireland
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Thomas Sejersen, MD

Karolinska Universitetssjukhuset
Stockholm, Sweden
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Sithara Ramdas

Oxford University hospitals NHS Foundation Trust
Oxford, United Kingdom
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Channa Hewamadduma

Royal Hallamshire Hospital
Sheffield, United Kingdom
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Recruitment Status: Open

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