(-)- Epicatechin Becker Muscular Dystrophy
| Status: | Completed |
|---|---|
| Conditions: | Neurology, Orthopedic |
| Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 18 - 70 |
| Updated: | 6/10/2018 |
| Start Date: | November 2016 |
| End Date: | December 2017 |
UCD0115B: An Open-label Extension Study of Purified Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy
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.
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.
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%
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