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

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

Condition or disease Intervention/treatment Phase
Becker Muscular Dystrophy Drug: (-)-epicatechin Phase 1 Phase 2

Detailed Description:

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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.
Study Start Date : May 2013
Estimated Primary Completion Date : January 2018
Estimated Study Completion Date : July 2018

Arm Intervention/treatment
Experimental: Treatment with Epicatechin
Purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.
Drug: (-)-epicatechin
purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.
Other Name: dietary supplement

Primary Outcome Measures :
  1. Muscle Function [ Time Frame: 8 weeks ]
  2. Muscle Stength [ Time Frame: 8 weeks ]

Secondary Outcome Measures :
  1. -(-)Epicatechin Pharmacokinetics [ Time Frame: 8 Weeks ]
    Standard clinical safety panel including: hematologic, hepatologic, renal and metabolic profiles

Other Outcome Measures:
  1. Normalization of Muscle Stucture [ Time Frame: 8 weeks ]
    Evaluation by histology, Western Blot, immunostain and electron microscopy

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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%

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01856868

Contact: Erica Goude, BA CCRP 916-734-0968

United States, California
University of California, Davis Recruiting
Sacramento, California, United States, 95817
Contact: Erica Goude, BA CCRP    916-734-0968   
Principal Investigator: Craig M McDonald, MD         
Sub-Investigator: Erik K Henricson, MPH         
Sub-Investigator: Richard T Abresch, MS         
Sub-Investigator: Frank Sharp, MD         
Sub-Investigator: Glenn Jickling, MD         
Sub-Investigator: Bjorn E Oskarsson, MD         
Sponsors and Collaborators
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

Additional Information:
Responsible Party: Craig McDonald, MD, Professor and Chairman, Department of Physical Medicine and Rehabilitation, University of California, Davis Identifier: NCT01856868     History of Changes
Other Study ID Numbers: 454352
First Posted: May 17, 2013    Key Record Dates
Last Update Posted: March 14, 2017
Last Verified: March 2017

Keywords provided by Craig McDonald, MD, University of California, Davis:
Becker muscular dystrophy
clinical trial
neuromuscular disease

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked