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Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

Primary Purpose

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Coenzyme Q10 and Lisinopril
Sponsored by
Cooperative International Neuromuscular Research Group
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Cardiac, Muscular Dystrophy, Randomized

Eligibility Criteria

8 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's National Medical Center
  • Lurie Children's Hospital
  • Carolinas Medical Center
  • University of Pittsburgh
  • University of Tennessee
  • Alberta Children's Hospital
  • National Center of Neurology and Psychiatry

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

No Intervention

Active Comparator

Active Comparator

Active Comparator

Arm Label

Enhanced standard of care

Lisinopril

Coenzyme Q10

Coenzyme Q10 and Lisinopril

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Full Information

First Posted
May 18, 2010
Last Updated
June 14, 2018
Sponsor
Cooperative International Neuromuscular Research Group
Collaborators
United States Department of Defense
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1. Study Identification

Unique Protocol Identification Number
NCT01126697
Brief Title
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
Official Title
PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
Study Type
Interventional

2. Study Status

Record Verification Date
June 2018
Overall Recruitment Status
Completed
Study Start Date
February 2010 (undefined)
Primary Completion Date
December 2017 (Actual)
Study Completion Date
December 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cooperative International Neuromuscular Research Group
Collaborators
United States Department of Defense

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Keywords
Cardiac, Muscular Dystrophy, Randomized

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Factorial Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
63 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Enhanced standard of care
Arm Type
No Intervention
Arm Title
Lisinopril
Arm Type
Active Comparator
Arm Title
Coenzyme Q10
Arm Type
Active Comparator
Arm Title
Coenzyme Q10 and Lisinopril
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
Coenzyme Q10 and Lisinopril
Intervention Description
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 Outcome Measure Information:
Title
myocardial performance index (MPI)
Description
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.
Time Frame
every 6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
Children's National Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Lurie Children's Hospital
City
Chicago
State/Province
Illinois
Country
United States
Facility Name
Carolinas Medical Center
City
Charlotte
State/Province
North Carolina
Country
United States
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
University of Tennessee
City
Memphis
State/Province
Tennessee
Country
United States
Facility Name
Alberta Children's Hospital
City
Calgary
State/Province
Alberta
Country
Canada
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
Country
Japan

12. IPD Sharing Statement

Links:
URL
http://www.cinrgresearch.org
Description
CINRG Network Website

Learn more about this trial

Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

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