Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

NCT00654784 | Completed Phase 2 Results

• 1 other identifier: SNT-II-001
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

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 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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

Duchenne Muscular Dystrophy; DMD; Duchenne

Outcome Measures

Primary Outcomes (1)

• The Relative Change in Peak Systolic Radial Strain of the Left Ventricle (LV) Inferolateral Wall From Baseline (at Screening) to Week 52, Assessed by Color Doppler Myocardial Imaging (CDMI).

  * Assessing the peak systolic radial strain of the left ventricle inferolateral wall is used to characterize the cardiac involvement in the DMD patients. * Color Doppler Myocardial Imaging technique is used to quantify regional myocardial function. The cardiac involvement in DMD is characterized by degeneration, atrophy and fibrosis of the myocardium, leading to dilated cardiomyopathy. The process begins in the posterolateral wall of the left ventricle, with septal involvement appearing at later stages.

  baseline and Week 52

Secondary Outcomes (3)

• Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF)

  1 year

• 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)

  1 year

• Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG.

  1 year

Study Arms (2)

1

EXPERIMENTAL

Drug: idebenone

2

PLACEBO COMPARATOR

Drug: placebo

Interventions

idebenone DRUG

idebenone 450 mg/day (150 mg three times a day)

1

placebo DRUG

2

Eligibility Criteria

• Age: 8 Years - 16 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)
• Ability to provide reproducible repeat quantitative muscle testing (QMT) upper limb score within 15% of first assessment score (at Visit1/Day 1 versus Screening Visit

You may not qualify if:

• Symptomatic cardiomyopathy or heart failure
• Asymptomatic but severe cardiac dysfunction on baseline (Screening) evaluation: Fractional shortening (FS) \< 20% and/or Ejection fraction (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
• Known individual hypersensitivity to idebenone

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (1)

Children's Hospital, University Hospital

Leuven, Belgium

Location

Related Publications (1)

• Buyse GM, Goemans N, van den Hauwe M, Thijs D, de Groot IJ, Schara U, Ceulemans B, Meier T, Mertens L. Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial. Neuromuscul Disord. 2011 Jun;21(6):396-405. doi: 10.1016/j.nmd.2011.02.016. Epub 2011 Mar 23.

  PMID: 21435876 RESULT

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

idebenone

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Thomas Meier
• Organization: Santhera

thomas.meier@santhera.com

+4161 906 8964

Study Officials

• Gunnar Buyse, MD PhD

  Universitaire Ziekenhuizen KU Leuven

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: April 3, 2008
• First Posted: April 9, 2008
• Study Start: October 1, 2005
• Primary Completion: August 1, 2007
• Study Completion: August 1, 2007
• Last Updated: August 1, 2011
• Results First Posted: July 29, 2011

Record last verified: 2011-07

Locations

BE (1)