Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

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

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.
    


Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

myocardial performance index (MPI)


Condition

Duchenne Muscular Dystrophy

Intervention

Coenzyme Q10 and Lisinopril

Study Arms / Comparison Groups

 Enhanced standard of care
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

63

Start Date

February 2010

Completion Date

December 2017

Primary Completion Date

December 2017

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
      

Gender

All

Ages

8 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT01126697

Organization ID

PITT0908


Responsible Party

Sponsor

Study Sponsor

Cooperative International Neuromuscular Research Group

Collaborators

 United States Department of Defense

Study Sponsor

, , 


Verification Date

June 2018