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

NCT ID: NCT01856868

Last Updated: 2021-12-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-05-31
• Study Completion Date: 2018-09-30

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 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.

Conditions

Becker Muscular Dystrophy

Keywords

BMD; Becker muscular dystrophy; epicatechin; clinical trial; neuromuscular disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment with Epicatechin

Purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.

Group Type: EXPERIMENTAL

(-)-epicatechin

Intervention Type: DRUG

purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.

Interventions

(-)-epicatechin

purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.

Intervention Type: DRUG

Other Intervention Names

dietary supplement

Eligibility 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%

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Cardero Therapeutics, Inc.

INDUSTRY

Sponsor Role: collaborator

Craig McDonald, MD

OTHER

Sponsor Role: lead

Responsible Party

Craig McDonald, MD

Professor and Chairman, Department of Physical Medicine and Rehabilitation

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Craig M McDonald, MD

Role: PRINCIPAL_INVESTIGATOR

University of California, Davis

Erik K Henricson, MPH

Role: STUDY_DIRECTOR

University of California, Davis

Locations

University of California, Davis

Sacramento, California, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Related Links

http://www.ucdmc.ucdavis.edu/pmr/research/Index.html

UC Davis Neuromuscular Research Center

Other Identifiers

454352

Identifier Type: -

Identifier Source: org_study_id