(-)- Epicatechin Becker Muscular Dystrophy
Condition: Becker Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: (-)-Epicatechin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 2.0
Sex: Male
Minimum age: 18 Years
Maximum age: 70 Years
Healthy volunteers: No
Keywords: BMD; Becker muscular dystrophy; epicatechin; clinical trial; neuromuscular disease

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%