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Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:
NCT01126697
First received: May 18, 2010
Last updated: October 10, 2014
Last verified: October 2014
  Purpose

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.


Condition Intervention Phase
Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Limb Girdle Muscular Dystrophy
Drug: Coenzyme Q10 and Lisinopril
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Factorial Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

Resource links provided by NLM:


Further study details as provided by Cooperative International Neuromuscular Research Group:

Primary Outcome Measures:
  • myocardial performance index (MPI) [ Time Frame: every 6 months ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 120
Study Start Date: February 2010
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
No Intervention: Enhanced standard of care
Active Comparator: Lisinopril Drug: Coenzyme Q10 and Lisinopril
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).
Active Comparator: Coenzyme Q10 Drug: Coenzyme Q10 and Lisinopril
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).
Active Comparator: Coenzyme Q10 and Lisinopril Drug: Coenzyme Q10 and Lisinopril
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).

  Eligibility

Ages Eligible for Study:   8 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01126697

Locations
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Illinois
Lurie Children's Hospital
Chicago, Illinois, United States
United States, North Carolina
Carolinas Medical Center
Charlotte, North Carolina, United States
United States, Pennsylvania
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Tennessee
University of Tennessee
Memphis, Tennessee, United States
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada
Japan
National Center of Neurology and Psychiatry
Tokyo, Japan
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group
  More Information

Additional Information:
No publications provided

Responsible Party: Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier: NCT01126697     History of Changes
Other Study ID Numbers: PITT0908
Study First Received: May 18, 2010
Last Updated: October 10, 2014
Health Authority: United States: Federal Government
United States: Institutional Review Board

Keywords provided by Cooperative International Neuromuscular Research Group:
Cardiac
Muscular Dystrophy
Randomized

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophies, Limb-Girdle
Muscular Dystrophy, Duchenne
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases
Coenzyme Q10
Lisinopril
Ubiquinone
Angiotensin-Converting Enzyme Inhibitors
Antihypertensive Agents
Cardiotonic Agents
Cardiovascular Agents
Enzyme Inhibitors
Growth Substances
Micronutrients
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Protease Inhibitors
Protective Agents
Therapeutic Uses
Vitamins

ClinicalTrials.gov processed this record on November 25, 2014