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

Craig McDonald, MD

Status and phase

Completed

Phase 2

Phase 1

Conditions

Becker Muscular Dystrophy

Treatments

Drug: (-)-epicatechin

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT01856868

454352

Details and patient eligibility

About

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

Full 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.

Enrollment

7 patients

Sex

Male

Ages

18 to 60 years old

Volunteers

No Healthy Volunteers

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%