Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

Sponsor

Cooperative International Neuromuscular Research Group (Other)

Overall Status

Completed

CT.gov ID

NCT01126697

Collaborator

United States Department of Defense (U.S. Fed)

Enrollment

Locations

Arms

Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

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.

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

Study Design

Study Type:

Interventional

Actual Enrollment :

63 participants

Allocation:

Randomized

Intervention Model:

Factorial Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Feb 1, 2010

Actual Primary Completion Date :

Dec 1, 2017

Actual Study Completion Date :

Dec 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
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).

Outcome Measures

Primary Outcome Measures

myocardial performance index (MPI) [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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

8 Years and Older

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's National Medical Center	Washington	District of Columbia	United States	20010
2	Lurie Children's Hospital	Chicago	Illinois	United States
3	Carolinas Medical Center	Charlotte	North Carolina	United States
4	University of Pittsburgh	Pittsburgh	Pennsylvania	United States	15213
5	University of Tennessee	Memphis	Tennessee	United States
6	Alberta Children's Hospital	Calgary	Alberta	Canada
7	National Center of Neurology and Psychiatry	Tokyo		Japan