Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by Cooperative International Neuromuscular Research Group
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:
NCT01126697
First received: May 18, 2010
Last updated: May 19, 2014
Last verified: May 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

Contacts
Contact: Lauren Hache, MS, CGC 412-224-2030 lhache@childrensnational.org
Contact: Andrea Smith, MS, CGC 412-383-7207 smithal7@upmc.edu

Locations
United States, California
University of California Los Angeles Recruiting
Los Angeles, California, United States
Contact: Lester Tsai       LTsai@mednet.ucla.edu   
Principal Investigator: Perry Shieh, MD         
University of California, Davis Recruiting
Sacramento, California, United States
Contact: Erica Goude, MS, CCRP    916-734-0968    pmr.research@ucdmc.ucdavis.edu   
Principal Investigator: Craig McDonald, MD         
Sub-Investigator: Erik Henricson, MPH         
United States, District of Columbia
Children's National Medical Center Recruiting
Washington, District of Columbia, United States, 20010
Contact: Bemnete Tadesse, BS    202-476-4802    btadesse@childrensnational.org   
Principal Investigator: Christopher Spurney, MD         
United States, Illinois
Lurie Children's Hospital Recruiting
Chicago, Illinois, United States
Contact: Lauren Webb    312-227-4483    lwebb@luriechildrens.org   
Principal Investigator: Nancy Kuntz, MD         
United States, North Carolina
Carolinas Medical Center Recruiting
Charlotte, North Carolina, United States
Contact: Jacqueline Sykes    704-333-3967    jacqueline.sykes@carolinas.org   
Principal Investigator: Susan Sparks, MD, PhD         
United States, Pennsylvania
University of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Andrea Smith, MS, CGC    412-383-7207    smithal7@upmc.edu   
Principal Investigator: Paula Clemens, MD         
United States, Tennessee
University of Tennessee Recruiting
Memphis, Tennessee, United States
Contact: Meegan Barrett-Adair    901-725-8920    mbarret9@uthsc.edu   
Principal Investigator: Tulio Bertorini, MD         
Canada, Alberta
Alberta Children's Hospital Recruiting
Calgary, Alberta, Canada
Contact: Jean Mah, MD       jean.mah@albertahealthservices.ca   
Principal Investigator: Jean Mah, MD         
Japan
National Center of Neurology and Psychiatry Recruiting
Tokyo, Japan
Contact: Maki Ohhata    81-42-341-2711    chiken5@ncnp.go.jp   
Principal Investigator: H Komaki, MD         
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: May 19, 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, Limb-Girdle
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Coenzyme Q10
Ubiquinone
Lisinopril
Micronutrients
Growth Substances
Physiological Effects of Drugs
Pharmacologic Actions
Vitamins
Angiotensin-Converting Enzyme Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antihypertensive Agents
Cardiovascular Agents
Therapeutic Uses
Cardiotonic Agents
Protective Agents

ClinicalTrials.gov processed this record on August 21, 2014