Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

The study will include 120 participants aged 8 and up with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies that have no clinical cardiac symptoms. Participants will be randomized to one of four arms: Arm 1 CoQ10 alone, Arm 2 Lisinopril alone, Arm 3 CoQ10 and Lisinopril or Arm 4 No study medication. Randomization will be stratified by ambulatory status and corticosteroid use. The primary outcome for the study is the myocardial performance index (MPI), measured by standard Doppler echocardiography. The study will last 24 months with visits at Months 0.5,1.5, 6, 12, 18 and 24.

Following completion of the Clinical Trial of Coenzyme Q10 and Lisinopril, participants will be offered participation in a companion protocol: PITT1215 A Natural History Companion Study to PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies. The objective of this study is to evaluate the longitudinal natural history of DMD, BMD, and LGMD2I and to evaluate the effects of Coenzyme Q10 and/or Lisinopril on prevention of cardiac dysfunction in these disorders.This will be an 18-month longitudinal natural history study designed to accompany the Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies.

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Key information

Conditions

Duchenne Muscular Dystrophy Becker Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Limb Girdle Muscular Dystrophy Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophies, Limb-Girdle Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

8 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Alberta Children's Hospital, Calgary, Alberta, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

8 years of age or older
Confirmed genetic diagnosis of Duchenne, Becker, or Limb Girdle muscular dystrophy
Beta-blocker naïve
Screening Doppler echocardiographic MPI measurement greater than or equal to 0.40 for the highest MPI value (spectral and tissue) or circumferential strain measured by STE that is less negative than or equal to - 23
Normal left ventricular fractional shortening (≥28%) and no clinical cardiac symptoms
Has not participated in other therapeutic research protocol within the last 6 months prior to screening
Ability to swallow tablets

Exclusion criteria

Spine curvature greater than 30% (based on the x-ray performed at screening)
History of significant concomitant illness or significant impairment of renal or hepatic function
History of hypersensitivity to ACE inhibitors
History of idiopathic or hereditary angioedema or a history of angioedema with prior ACE inhibitor use
Use of carnitine, creatine, glutamine, or any herbal medicines (this would not include herbal teas unless they are consumed daily with intended medicinal effect) in the 3-months prior to enrollment
CoQ10 and/or ACE inhibitor use for a duration greater than 6 months
CoQ10 and/or ACE inhibitor use in the 3-months prior to enrollment
CoQ10 serum level of 2.5 ug/ml or higher
Investigator assessment of inability to comply with protocol

Treatment and study plan

Coenzyme Q10 and Lisinopril

Drug

Arm 1. Coenzyme Q10: taken once a day each morning by mouth OR Arm 2. Lisinopril: taken once a day each morning by mouth OR Arm 3. Coenzyme Q10 and lisinopril: each taken once a day in the morning by mouth OR Arm 4. Enhanced Standard Care (more doctor visits, muscle and breathing testing, and x-rays for monitoring, but no study medication).

Primary outcomes

myocardial performance index (MPI)

Time frame: every 6 months

The MPI is a sensitive, quantifiable, noninvasive measure of global ventricular function that is independent of cardiac geometry and heart rate. MPI is collected through standard echocardiogram assessment. MPI is a ratio of the total time spent in isovolumic activity (isovolumic contraction time and isovolumic relaxation time) to the time spent in ventricular ejection.

Sponsors and collaborators

Lead sponsor

Cooperative International Neuromuscular Research Group

Network

Collaborators

United States Department of Defense

Registry information

Official study title

PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

Important dates

Study start: 2010
Primary completion: 2017
Study completion: 2017
First posted: May 20, 2010
Registry last updated: Jun 15, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

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