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

Completed · Phase 3 · Has a placebo group

Conditions studied: Muscular Dystrophy, Duchenne, Ambulatory Care

In brief

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.

Key facts

Study ID

NCT01027884

Run by

Santhera Pharmaceuticals

People needed

65

Starts

2009-07-01

Expected to finish

2014-04-01

Last updated by the study team

2015-10-19

Who can join

Age: 10 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Patients 10 - 18 years of age at Baseline.
• Signed and dated informed consent.
• Documented diagnosis of DMD or severe dystrophinopathy and clinical features consistent of typical DMD at diagnosis (i.e. documented delayed motor skills and muscle weakness by age 5 years). DMD should be confirmed by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostain.
• Ability to provide reliable and reproducible repeat PEF within 15% of the first assessment (i.e. Baseline vs. Screening).
• Patients assessed by the investigator as willing and able to comply with the requirements of the study, possess the required cognitive abilities and are able to swallow study medication.

You may not qualify if…

• Patients dependent on assisted ventilation at Screening and/or Baseline (defined as non-invasive nocturnal ventilation, daytime non-invasive ventilation or continuous invasive ventilation).
• Patients with documented DMD-related hypoventilation for which assisted ventilation is needed according to current standard of care guidelines (e.g. FVC< 30%) or is required in the opinion of the Investigator.
• Patients with a percent predicted PEF > 80% at Baseline.
• Patients unable to form a mouth seal to allow precise respiratory flow measurements and mouth pressures.
• Symptomatic heart failure (high probability of death within one year of Baseline) and/or symptomatic ventricular arrhythmias.
• Participation in the previous Phase II or Phase II Extension study (SNT-II-001 or SNT-II-001-E) for idebenone.
• Participation in any other therapeutic trial and/or intake of any investigational drug within 90 days prior to Baseline.
• Use of carnitine, creatine, glutamine, oxatomide, or any herbal medicines within 30 days prior to Baseline.
• Use of coenzyme Q10 or vitamin E (if taken at a dose of 5 times above the daily physiological requirement) within 30 days prior to Baseline.
• Any previous use of idebenone.
• Any concomitant medication with a depressive or stimulating effect on respiration or the respiratory tract.
• Planned or expected spinal fixation surgery during the study period (as judged by the investigator).
• Asthma, bronchitis/COPD, bronchiectasis, emphysema, pneumonia or the presence of any other non-DMD respiratory illness that affects PEF.
• Chronic use of beta-2 agonists or any use of other bronchodilating medication (e.g. inhaled steroids, sympathomimetics, anticholinergics).
• Please note: Chronic use if defined as a daily intake for more than 14 days.
• Moderate or severe hepatic impairment or severe renal impairment.
• Prior or ongoing medical condition or laboratory abnormality that in the Investigator's opinion could adversely affect the safety of the subject.
• Please note: Patients who suffer from a severe, unstable condition including (but not limited to) cancer, auto-immune diseases, haematological diseases, metabolic disorders or immunodeficiencies, and who are at risk of an aggravation unrelated to the study condition, can only be included in the study if accepted in writing by the Sponsor's Medical Monitor.
• Relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana products/smoking
• Known individual hypersensitivity to idebenone or to any of the ingredients/excipients of the study medication
• Systemic glucocorticoid therapy
• Chronic use of systemic glucocorticoid therapy for DMD related conditions within 12 months of Baseline (the "12 month non-use period")
• More than 2 rounds of acute systemic glucocorticoid burst therapy (of ≤2 week duration) for non-DMD related conditions within the 12 month non-use period
• Use of any round of systemic glucocorticoid burst therapy of longer than 2 weeks duration within the 12 month non-use period
• Use of systemic glucocorticoid burst therapy less than 8 weeks prior to baseline

Where it is running

• University of California Davis Medical Center — Sacramento, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• University of Florida — Gainesville, Florida, United States
• Carolinas Medical Center, Neurosciences and Spine Institute — Charlotte, North Carolina, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Monroe Carell, Jr. Children's Hospital at Vanderbilt — Nashville, Tennessee, United States
• University of Texas Southwestern Medical Center — Dallas, Texas, United States
• Methodist Neurological Institute — Houston, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Dr. Günther Bernert, Prim. Univ. Doz. — Vienna, Austria
• University Hospitals Leuven- Children Hospital — Leuven, Belgium
• Hôpital Roger Salengro, CHRU Lille — Lille, France
• Prof. Thomas Voit , MD, PhD — Paris, France
• Universitätsklinikum Essen, Zentrum für Kinderheikunde — Essen, Germany
• Universitätsklinik Freiburg Zentrum für Kinderheilkunde und Jugendmedizin — Freiburg im Breisgau, Germany
• Fondazione IRCCS "Eugenio Medea" — Bosisio Parini, Lecco, Italy
• Azienda Ospedaliera Niguarda Ca' Granda Centro Clinico Nemo — Milan, Italy
• Azienda Ospedaliera Universitaria della Seconda Università degli Studi di Napoli — Naples, Italy
• Ass. Prof. Jan Verschuuren , MD, PhD — Leiden, P.O. Box 9600, Netherlands
• Hospital Universitario 12 de Octubre — Madrid, Spain
• Hospital Universitario y Politécnico La Fe — Valencia, Spain
• Prof. Thomas Sejersen, MD, PhD — Stockholm, Sweden
• CHUV Lausanne Neuropediatrie — Lausanne, Switzerland

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.