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

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.

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%