Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

Summary

The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD. Furthermore, the safety and tolerability of idebenone was assessed.

Conditions

• Muscular Dystrophy, Duchenne
• Ambulatory Care

Interventions

DRUG

Placebo

Placebo (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals

DRUG

Idebenone

Idebenone (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals

Sponsors & Collaborators

• Santhera Pharmaceuticals

  lead INDUSTRY

Principal Investigators

• Prof. Gunnar Buyse, MD, PhD. · University Hospitals Leuven, B-3000, Belgium

• Dr. Ulrike Schara, MD, PhD · Universitätsklinikum Essen, D-45122 Essen, Germany

• Ass. Prof. Jan Verschuuren, MD, PhD · Leiden University Medical Center (LUMC), 2300 RC Leiden, the Netherlands

• Dr. Pierre-Yves Jeannet, Médecin Associé, MER · Unité de Neuropédiatrie, CHUV - BH11, 1011 Lausanne-CH, Switzerland

• Prof. Thomas Voit, MD, PhD · Université Pierre et Marie curie VI - Institut de Myologie - groupe hospitalier Pitié Salpétrière - 47/83 boulevard de l'hôpital, 75651 Paris Cedex 13, France

• Prof. Thomas Sejersen, MD, PhD · Astrid Lindgrens Barnsjukhus- Karolinska Universitetssjukhuset, SE-17176 Stockholm, Sweden

• Dr. Günther Bernert, Prim. Univ. Doz. · Vorstand der Abteilung für Kinder- und Jugendheilkunde, Gottfried v. Preyer'sches Kinderspital, 1100 Wien, Austria

• Gihan Tennekoon, MD · Division of Neurology - The Children's Hospital of Philadelphia - 34th Street and Civic Center Blvd, Philadelphia, PA 19104-1771, USA

• Jean-Marie Cuisset, MD · Hôpital Roger Salengro, CHRU, Service de neurologie infantile, Lille, France

• Susan Iannaccone, MD · University of Texas Southwestern Medical Center, TX, USA

• Susan Sparks, MD · The Charlotte-Mecklenburg Hospital Authority, Charlotte, NC, USA

• Janbernd Kirschner, MD · Universitätsklinik Freiburg Zentrum für Kinderheilkunde und Jugendmedizin

• Maria Grazia Nadia D'Angelo, MD · Fondazione IRCCS "Eugenio Medea"

• Ksenija Gorni, MD · Azienda Ospedaliera Niguarda Ca'Granda Centro Clinico Nemo

• Bryan W. Burnette, MD · Monroe Carell Jr. Children's Hospital at Vanderbilt

• Barry Byrne, MD · University of Florida

• Michele Yang, MD · Children's Hospital Colorado

• Susan Apkon, MD · Seattle Children's Hospital

• Ericka Simpson, MD · Methodist Neurological Institute, Houston

• Craig McDonald, MD · University of California, Davis

• Luisa Politano, MD · Azienda Ospedaliera Universitaria della Seconda Università degli Studi di Napoli

• Ana Camacho Salas, MD · Hospital Universitario 12 de Octubre

• Juan Jesus Vilchez, MD · Hospital Universitari y Politècnic La Fe de Valencia

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

10 Years

Max Age

18 Years

Sex

MALE

Healthy Volunteers

No

Timeline & Regulatory

Start

2009-07-31

Primary Completion

2014-01-31

Completion

2014-04-30

Countries

• United States
• Austria
• Belgium
• France
• Germany
• Italy
• Netherlands
• Spain
• Sweden
• Switzerland

Study Locations