Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy - Full Text View

Sponsored by:	Santhera Pharmaceuticals
Information provided by:	Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00654784

Purpose

Idebenone is a synthetic analogue of coenzyme Q10 and is a powerful antioxidant and essential constituent of the process of energy production on the cellular level. It can protect mitochondria (the powerplants of the body's cells) from oxidative damage and boost their impaired function. It is thought that this mechanism will slow decline in heart function that is part of the disease process of Duchenne Muscular Dystrophy (DMD). It is possible that patients may benefit in terms of muscle strength and respiratory function. This pilot trial is designed to investigate this.

Condition	Intervention	Phase
Duchenne Muscular Dystrophy (DMD)	Drug: idebenone Drug: placebo	Phase II

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy L1 syndrome

MedlinePlus related topics: Muscular Dystrophy

Drug Information available for: CV 2619

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title:	A Phase IIa Double Blind, Randomised, Placebo Controlled, Single Centre Study at the University of Leuven to Assess the Efficacy and Tolerability of Idebenone in 8 - 16 Year Old Males With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:

The relative change from Baseline (at Screening) to Week 52 in peak systolic radial strain of the LV inferolateral wall, assessed by Color Doppler Myocardial Imaging (CDMI). [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Respiratory Function: forced vital capacity (FVC), forced expiratory volume in 1 second (FEV1), maximal inspiratory pressure (MIP) and peak flow (PF) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Skeletal muscle strength (upper limb, right and left): hand grip, elbow flexors and elbow extensors (upper limb score) Timed walking test (10 metres) (ambulant patients only) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Safety and tolerability, assessed by adverse events, blood and urine laboratory measures, ECG. [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]

Enrollment:	21
Study Start Date:	October 2005
Study Completion Date:	August 2007
Primary Completion Date:	August 2007 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental	Drug: idebenone idebenone 450 mg/day (150 mg three times a day)
2: Placebo Comparator	Drug: placebo

Eligibility

Ages Eligible for Study:	8 Years to 16 Years
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patients 8 - 16 years of age at time of enrolment
Male
Presence of cardiac involvement/dysfunction, defined by abnormal peak systolic strain in left ventricle (LV) inferolateral wall
Confirmed diagnosis of DMD (out of frame dystrophin gene deletion OR absent/<5% dystrophin protein on muscle biopsy; clinical picture consistent of typical DMD)
If on chronic glucocorticosteroids treatment (deflazacort, prednisone) for DMD (or any other disease) (i.e. concomitant medication): dosage must be stable (unchanged) 6 months prior to inclusion
If on chronic medication for DMD associated cardiomyopathy (β-blocker, diuretics): dosage must be stable (unchanged) 3 months prior to inclusion
Ability to provide reproducible repeat QMT upper limb score within 15% of first assessment score (at Visit1/Day 1 versus Screening Visit

Exclusion Criteria:

Symptomatic cardiomyopathy or heart failure
Asymptomatic but severe cardiac dysfunction on baseline (Screening) evaluation: FS < 20% and/or EF < 40%
Use of ACE-inhibitors
Previous history of ventricular arrhythmias (other than isolated ventricular extrasystole); ventricular arrhythmias presented at Screening
Previous (6 months or less) participation in any other therapeutic trial for DMD
Use of coenzymeQ10, idebenone, creatine, glutamine, oxatomide, or any herbal medicines within the last 6 months
History of significant concomitant illness or significant impairment of renal or hepatic function (serum creatinine and GGT greater than 1.5 times upper limit for age and gender)
Known individual hypersensitivity to idebenone

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00654784

Locations

Belgium
Children's Hospital, University Hospital
Leuven, Belgium

Sponsors and Collaborators

Santhera Pharmaceuticals

Investigators

Principal Investigator:

Gunnar Buyse, MD PhD

University Hospital Leuven

More Information

No publications provided

Study ID Numbers:	SNT-II-001
Study First Received:	April 3, 2008
Last Updated:	December 10, 2008
ClinicalTrials.gov Identifier:	NCT00654784 History of Changes
Health Authority:	Belgium: The Federal Public Service (FPS) Health, Food Chain Safety and Environment

Keywords provided by Santhera Pharmaceuticals:

Duchenne Muscular Dystrophy
DMD
Duchenne

Study placed in the following topic categories:

Becker's Muscular Dystrophy
Antioxidants
Idebenone
Muscular Dystrophy, Duchenne and Becker Type
Muscular Dystrophies
Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases

Neuromuscular Diseases
Genetic Diseases, Inborn
Muscular Dystrophy, Duchenne
Duchenne Muscular Dystrophy
Genetic Diseases, X-Linked
Atrophy
Muscular Dystrophy

Additional relevant MeSH terms:

Antioxidants
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Nervous System Diseases
Idebenone
Protective Agents
Pharmacologic Actions
Muscular Dystrophies

Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Muscular Dystrophy, Duchenne
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on May 07, 2009