Clinical Trial: Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: An Open-label Pilot Study of Purified Tea-derived Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy

Brief Summary: (-)-Epicatechin will be evaluated for the treatment of progressive muscle loss and impaired skeletal muscle function in Becker Muscular Dystrophy (BMD) patients.

Detailed Summary:

This is a proof-of-concept phase 1/2a pilot and endpoint development study that is designed to provide initial evidence of biological activity of (-)-epicatechin. Primary endpoints include initial assessment of tissue-specific evidence of efficacy from muscle biopsy samples. Secondary endpoints include measures of strength and physical function, and safety and adverse event data. Pilot endpoints include assessment of mRNA and miRNA peripheral blood profiles and validation of non-invasive near-infrared spectroscopy (NIRS) muscle perfusion studies during exercise and a recumbent cycle exercise test that may be employed as endpoints in future clinical trials.

This single center open-label pilot study will enroll 10 adults with genetically-confirmed Becker muscular dystrophy, who will receive the purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks. After screening visits, participants will be enrolled in the study if they meet all inclusion criteria. They will be evaluated at baseline and at screening, day 1, and weeks 1, 2, 4 and 8.


Sponsor: Craig McDonald, MD

Current Primary Outcome:

  • Muscle Function [ Time Frame: 8 weeks ]
  • Muscle Stength [ Time Frame: 8 weeks ]


Original Primary Outcome: Efficacy Primary Endpoint: Evaluation of (-)-epicatechin on blood and muscle tissue markers of mitochondrial biogenesis and muscle regeneration in adults with Becker muscular dystrophy. [ Time Frame: 8 weeks ]

Treatment with 8 weeks of (-)- epicatechin 100mg daily will induce mitochondrial biogenesis, muscle regeneration, and improved histological appearance in sarcomere morphology.


Current Secondary Outcome: -(-)Epicatechin Pharmacokinetics [ Time Frame: 8 Weeks ]

Standard clinical safety panel including: hematologic, hepatologic, renal and metabolic profiles


Original Secondary Outcome:

  • Efficacy Secondary Endpoints: Evaluation of the effects of (-)-epicatechin on exercise capacity in adults with Becker muscular dystrophy. [ Time Frame: 8 Weeks ]
    Individuals with Becker muscular dystrophy who receive 100mg/day of (-)-epicatechin will demonstrate improved exercise performance and strength that is associated with increases in mitochondrial biogenesis and muscle regeneration.
  • Safety Secondary Endpoints: Evaluation of safety and pharmacokinetic profiles of (-)-epicatechin in adults with Becker muscular dystrophy. [ Time Frame: 8 Weeks ]
    Assessments of safety will include a standard clinical safety panel including hematologic, hepatologic, renal and metabolic profiles. Pharmacokinetic studies will include repeat assessments of trough, 2 hour-post (peak) and 4-hour post dose (-)-epicatechin levels.


Information By: University of California, Davis

Dates:
Date Received: May 9, 2013
Date Started: May 2013
Date Completion: July 2018
Last Updated: March 10, 2017
Last Verified: March 2017