Phase 2 N=2 Treatment

(-)- Epicatechin Becker Muscular Dystrophy

Becker Muscular Dystrophy

Bottom Line

View on ClinicalTrials.gov: NCT03236662 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Jul 2021

Primary outcome: Primary: Plasma Follistatin

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: (-)-Epicatechin (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: Male
Sponsor: Craig McDonald, MD
Primary completion: Nov 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Plasma Follistatin	—	—
PRIMARY Plasma Myostatin	—	—
PRIMARY Plasma Nitrates/ SNO	—	—
PRIMARY Plasma BNP	—	—
PRIMARY Plasma Creatine Kinase	—	—
PRIMARY Plasma MMP-9	—	—
PRIMARY Plasma TNF-Alpha	—	—
PRIMARY Plasma TGF-Beta	—	—
PRIMARY Plasma Follistatin:Myostain Ratio	—	—
SECONDARY Graded Exercise Test Using a Recumbent Cycle Ergometer	—	—
SECONDARY 6-minute Walk Test	—	—

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.

Eligibility Criteria

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%

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03236662). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.