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

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2013 by University of California, Davis
Sponsor:
Collaborator:
Cardero Therapeutics, Inc.
Information provided by (Responsible Party):
Craig McDonald, MD, University of California, Davis
ClinicalTrials.gov Identifier:
NCT01856868
First received: May 9, 2013
Last updated: August 23, 2013
Last verified: August 2013
  Purpose

(-)-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.


Condition Intervention Phase
Becker Muscular Dystrophy
Drug: (-)-epicatechin
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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.

Resource links provided by NLM:


Further study details as provided by University of California, Davis:

Primary Outcome Measures:
  • Muscle Function [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Muscle Stength [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • -(-)Epicatechin Pharmacokinetics [ Time Frame: 8 Weeks ] [ Designated as safety issue: Yes ]
    Standard clinical safety panel including: hematologic, hepatologic, renal and metabolic profiles


Other Outcome Measures:
  • Normalization of Muscle Stucture [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
    Evaluation by histology, Western Blot, immunostain and electron microscopy


Estimated Enrollment: 10
Study Start Date: May 2013
Estimated Study Completion Date: May 2014
Estimated Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment Drug: (-)-epicatechin

  Eligibility

Ages Eligible for Study:   18 Years to 60 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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%
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01856868

Contacts
Contact: Erica Goude, BA CCRP 916-734-0968 erica.goude@ucdmc.ucdavis.edu

Locations
United States, California
University of California, Davis Recruiting
Sacramento, California, United States, 95817
Contact: Erica Goude, BA CCRP    916-734-0968    erica.goude@ucdmc.ucdavis.edu   
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.
Investigators
Principal Investigator: Craig M McDonald, MD University of California, Davis
Study Director: Erik K Henricson, MPH University of California, Davis
  More Information

Additional Information:
No publications provided

Responsible Party: Craig McDonald, MD, Professor and Chairman, Department of Physical Medicine and Rehabilitation, University of California, Davis
ClinicalTrials.gov Identifier: NCT01856868     History of Changes
Other Study ID Numbers: UCD0113
Study First Received: May 9, 2013
Last Updated: August 23, 2013
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration

Keywords provided by University of California, Davis:
BMD
Becker muscular dystrophy
epicatechin
clinical trial
neuromuscular disease

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

ClinicalTrials.gov processed this record on August 26, 2014