Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

NCT01126697 | Completed Phase 2 DMC

• 1 other identifier: PITT0908
• Study Type: interventional
• Target: 63
• Locations: 3 countries
• Sites: 7

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy; Becker Muscular Dystrophy; Limb Girdle Muscular Dystrophy

Keywords

Cardiac; Muscular Dystrophy; Randomized

Outcome Measures

Primary Outcomes (1)

• myocardial performance index (MPI)

  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.

  every 6 months

Study Arms (4)

Enhanced standard of care

NO INTERVENTION

Lisinopril

ACTIVE COMPARATOR

Drug: Coenzyme Q10 and Lisinopril

Coenzyme Q10

ACTIVE COMPARATOR

Drug: Coenzyme Q10 and Lisinopril

Coenzyme Q10 and Lisinopril

ACTIVE COMPARATOR

Drug: Coenzyme Q10 and Lisinopril

Interventions

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).

Coenzyme Q10; Coenzyme Q10 and Lisinopril; Lisinopril

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cooperative International Neuromuscular Research Group: lead
• United States Department of Defense: collaborator

Study Sites (7)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Lurie Children's Hospital

Chicago, Illinois, United States

Location

Carolinas Medical Center

Charlotte, North Carolina, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Tennessee

Memphis, Tennessee, United States

Location

Alberta Children's Hospital

Calgary, Alberta, Canada

Location

National Center of Neurology and Psychiatry

Tokyo, Japan

Location

Related Links

• CINRG Network Website

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies, Limb-Girdle; Muscular Dystrophies

Interventions

coenzyme Q10; Lisinopril

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Dipeptides; Oligopeptides; Peptides; Amino Acids, Peptides, and Proteins

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: FACTORIAL
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 18, 2010
• First Posted: May 20, 2010
• Study Start: February 1, 2010
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2017
• Last Updated: June 15, 2018

Record last verified: 2018-06

Locations

CA (1); JP (1); US (5)