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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01856868
Recruitment Status : Recruiting
First Posted : May 17, 2013
Last Update Posted : March 14, 2017
Cardero Therapeutics, Inc.
Information provided by (Responsible Party):
Craig McDonald, MD, University of California, Davis

May 9, 2013
May 17, 2013
March 14, 2017
May 2013
January 2018   (Final data collection date for primary outcome measure)
  • Muscle Function [ Time Frame: 8 weeks ]
  • Muscle Stength [ Time Frame: 8 weeks ]
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.
Complete list of historical versions of study NCT01856868 on Archive Site
-(-)Epicatechin Pharmacokinetics [ Time Frame: 8 Weeks ]
Standard clinical safety panel including: hematologic, hepatologic, renal and metabolic profiles
  • 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.
Normalization of Muscle Stucture [ Time Frame: 8 weeks ]
Evaluation by histology, Western Blot, immunostain and electron microscopy
Pilot Biomarker Endpoints: Pilot evaluation of disease- and epicatechin-specific circulating mRNA and miRNA blood profiles as pilot biomarkers for monitoring treatment efficacy. [ Time Frame: 8 weeks ]
Epicatechin stimulates follistatin expression in tissue and blood. In addition to its effects on muscle regeneration, follistatin is known to exert anti-inflammatory and anti-fibrotic effects via antagonism of activin and myostatin.
Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
An Open-label Pilot Study of Purified Tea-derived Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy.
(-)-Epicatechin will be evaluated for the treatment of progressive muscle loss and impaired skeletal muscle function in Becker Muscular Dystrophy (BMD) patients.

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.

Phase 1
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Becker Muscular Dystrophy
Drug: (-)-epicatechin
purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.
Other Name: dietary supplement
Experimental: Treatment with Epicatechin
Purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.
Intervention: Drug: (-)-epicatechin
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
July 2018
January 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male
  • Age 18 years to 60 years
  • Average to low daily physical activity
  • Ability to ambulate for 75 meters without assistive devices
  • Diagnosis of BMD confirmed by at least one the following:

    • Dystrophin immunofluorescence and/or immunoblot showing partial dystrophin deficiency, and clinical picture consistent with typical BMD, or
    • Gene deletions test positive (missing one or more exons) of the dystrophin gene, where reading frame can be predicted as 'in-frame', and clinical picture consistent with typical BMD, or
    • Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) that can be definitely associated with BMD, with a typical clinical picture of BMD, or
    • Positive family history of BMD confirmed by one of the criteria listed above in a sibling or maternal uncle, and clinical picture typical of BMD.
  • Nutritional, herbal and antioxidant supplements taken with the intent of maintaining or improving skeletal muscle strength or functional mobility have been discontinued at least 2 weeks prior to screening (daily multivitamin use is acceptable).
  • Hematology profile within normal range
  • Baseline laboratory safety chemistry profile within normal range
  • No plan to change exercise regimen during study participation

Exclusion Criteria:

  • Currently enrolled in another treatment clinical trial.
  • History of significant concomitant illness or significant impairment of renal or hepatic function.
  • Use of regular daily aspirin or other medication with antiplatelet effects within 3 weeks of first dose of study medication.
  • Regular participation in vigorous exercise.
  • Symptomatic heart failure with cardiac ejection fraction <25%
Sexes Eligible for Study: Male
18 Years to 60 Years   (Adult)
Contact: Erica Goude, BA CCRP 916-734-0968
United States
Not Provided
Not Provided
Not Provided
Craig McDonald, MD, University of California, Davis
Craig McDonald, MD
Cardero Therapeutics, Inc.
Principal Investigator: Craig M McDonald, MD University of California, Davis
Study Director: Erik K Henricson, MPH University of California, Davis
University of California, Davis
March 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP