(-)- Epicatechin Becker Muscular Dystrophy

Study Overview
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Brief Summary
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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.

Official Title
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UCD0115B: An Open-label Extension Study of Purified Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy

Conditions
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Becker Muscular Dystrophy

Intervention / Treatment
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* Drug: (-)-Epicatechin


Participation Criteria
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Eligibility Criteria
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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%

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment<br>(-)-epicatechin 50mg twice per day (100mg per day total dose) | Drug: (-)-Epicatechin<br> <br> |

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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BMD, Becker muscular dystrophy, epicatechin, clinical trial, neuromuscular disease