Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

Status:	Completed
Conditions:	Neurology, Orthopedic
Therapuetic Areas:	Neurology, Orthopedics / Podiatry
Healthy:	No
Age Range:	8 - Any
Updated:	6/17/2018
Start Date:	February 2010
End Date:	December 2017

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

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.

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

We found this trial at

sites

Childrens National Medical Center

111 Michigan Ave NW
Washington, District of Columbia

(202) 476-5000