Clinical trial NCT03236662: (-)- Epicatechin Becker Muscular Dystrophy
Official title: UCD0115B: An Open-label Extension Study of Purified Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Becker Muscular Dystrophy
Keywords: BMD
Becker muscular dystrophy
epicatechin
clinical trial
neuromuscular disease
Lead sponsor: University of California, Davis
Sponsor class: OTHER
Collaborators: name: Cardero Therapeutics, Inc.
First posted: 2017-08-02
Enrollment: 2
Sex: MALE
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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%

Primary outcomes:
measure: Plasma Follistatin
timeFrame: 48 weeks
measure: Plasma Myostatin
timeFrame: 48 weeks
measure: Plasma Nitrates/ SNO
timeFrame: 48 weeks
measure: Plasma BNP
timeFrame: 48 weeks
measure: Plasma Creatine Kinase
timeFrame: 48 weeks
measure: Plasma MMP-9
timeFrame: 48 weeks
measure: Plasma TNF-Alpha
timeFrame: 48 weeks
measure: Plasma TGF-Beta
timeFrame: 48 weeks
measure: Plasma Follistatin:Myostain Ratio
timeFrame: 48 weeks
Secondary outcomes:
measure: Graded Exercise Test Using a Recumbent Cycle Ergometer
timeFrame: baseline and at 2-minute intervals
measure: 6-minute Walk Test
timeFrame: 48 weeks
Other outcomes:
measure: Exploratory Proteomics
timeFrame: 48 weeks
Locations:
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
Study officials:
References: