(-)- Epicatechin Becker Muscular Dystrophy

Phase 2

Completed

Brief Summary: This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial. This single center study will enroll up to 10 adults 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 screening, baseline, and weeks 4, 8, 12, 24, 16 and 48. The main criterion for success of the study will be presence of one or more biologic or strength and performance outcome measures that yield a response magnitude that allows for sufficient power in a Phase II B study with a sample size of 30 individuals.

Recruitment & Eligibility

Inclusion Criteria

Prior participation in UCD0113 BMD epicatechin pilot study
Male
Age 18 years to 70 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.
Hematology profile within normal range
Baseline laboratory safety chemistry profile within normal range
No plan to change exercise regimen during study participation
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).

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%

Arm && Interventions

Group	Intervention	Description
Treatment	(-)-Epicatechin	(-)-epicatechin 50mg twice per day (100mg per day total dose)

Primary Outcome Measures

Name	Time	Method
Plasma BNP	48 weeks	blood biomarker concentration
Plasma Follistatin	48 weeks	blood biomarker concentration
Plasma Myostatin	48 weeks	blood biomarker concentration
Plasma Nitrates/ SNO	48 weeks	blood biomarker concentration
Plasma TGF-Beta	48 weeks	blood biomarker concentration
Plasma Creatine Kinase	48 weeks	blood biomarker concentration
Plasma MMP-9	48 weeks	blood biomarker concentration
Plasma TNF-Alpha	48 weeks	blood biomarker concentration
Plasma Follistatin:Myostain Ratio	48 weeks	Ratio of plasma follistatin to plasma myostatin

Secondary Outcome Measures

Name	Time	Method
Graded Exercise Test Using a Recumbent Cycle Ergometer	baseline and at 2-minute intervals	blood lactate measured
6-minute Walk Test	48 weeks	Measurements recorded will include 25-meter split times and total distance traveled.