CompletedNCT03236662Updated Nov 24, 2021Results posted

(-)- Epicatechin Becker Muscular Dystrophy

A Phase 2 interventional study of (-)-Epicatechin in Becker Muscular Dystrophy, sponsored by Craig McDonald, MD. Completed at 1 site in United States. Open to male participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2021-11-24.

Sponsored by Craig McDonald, MD · Phase 2, Interventional, and Treatment

Phase: Phase 2
Study type: Interventional
Enrollment: 2
Allocation: Not applicable
Ages: 18 Years to 70 Years
Sex: Male

Study 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.

Conditions studied

Becker Muscular Dystrophy

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Keywords

BMD
Becker muscular dystrophy
epicatechin
clinical trial
neuromuscular disease

In context

Muscular Dystrophies

548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.

This study's enrollment of 2 is below the median of 24 across 344 interventional studies indexed under Muscular Dystrophies.

Browse Muscular Dystrophies studies →

Lead sponsor

Craig McDonald, MD is the lead sponsor of 3 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years to 70 Years
Sexes eligible: Male
Accepts healthy volunteers: No

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

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%

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 2 participants (actual)

Study arms

Experimental
Treatment
(-)-epicatechin 50mg twice per day (100mg per day total dose)
Drug: (-)-Epicatechin

Interventions

Drug(-)-Epicatechin

What researchers measure

Primary outcomes

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

Secondary outcomes

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

Other outcomes

Exploratory Proteomics
Collection of plasma samples for proteomics analysis.
Time frame: 48 weeks

Results

Posted Jul 26, 2021

Limitations and caveats

Small number of subjects analyzed.

Participant flow

Participant flow — Overall Study
Milestone	Treatment
Started	2
Completed	2
Not completed	0