Status:
COMPLETED
Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy
Lead Sponsor:
Epirium Bio Inc.
Conditions:
Becker Muscular Dystrophy
Eligibility:
MALE
16-59 years
Phase:
PHASE1
Brief Summary
This is a Phase 1, open-label, dose escalation study aimed at evaluating the safety, early efficacy and potential biomarkers of (+)-epicatechin in patients with Becker or Becker-like Muscular Dystroph...
Detailed Description
The safety and tolerability of three escalating doses of (+)-epicatechin will be assessed and early effectiveness measured by changes in plasma biomarkers, tissue biomarkers from muscle biopsies, card...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Participant must be ≥16 to \<60 years of age.
- Genotype confirmation showing a mutation of the dystrophin gene.
- Ambulation - participants must show a history of ambulation past the age of 16 years, with continued ambulation thereafter.
- If on glucocorticoid treatment in the last 12 months, participants must be on a stable dose at screening. Participants cannot start steroids during the study.
- EXCLUSION CRITERIA:
- A diagnosis of other neurological diseases or presence of relevant somatic disorders that are not related to Becker muscular dystrophy.
- Participants with a history of migraine headaches requiring medical attention and active treatment within the past 6 months.
- Participants with allergies to chocolate or cocoa.
- Surgery or orthopedic injury that might affect muscle strength or function within 3 months before study entry or planned surgery at any time during the study.
- Presence of a concomitant neurologic disease (e.g., Parkinson's disease) that could negatively impact mobility or balance.
- Symptomatic heart failure (New York Heart Association Class III or IV) or known left ventricular ejection fraction \<40% by echocardiogram.
- Presence of documented intrinsic lung disease (e.g., chronic obstructive pulmonary disease, pulmonary fibrosis).
- Evidence of current liver disease or impairment.
- Inadequate renal function.
- Platelet count, WBC count, and hemoglobin at Screening \<Lower Limit of Normal (LLN).
- Surgery or orthopedic injury that might affect muscle strength or function within 3 months before study entry or planned surgery at any time during the study
Exclusion
Key Trial Info
Start Date :
July 13 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2022
Estimated Enrollment :
22 Patients enrolled
Trial Details
Trial ID
NCT04386304
Start Date
July 13 2020
End Date
March 1 2022
Last Update
March 23 2022
Active Locations (3)
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1
UCLA Dept of Human Genetics
Los Angeles, California, United States, 90095
2
University of California - Davis Department of Physical Medicine and Rehabilitation
Sacramento, California, United States, 95817
3
Washington University School of Medicine
St Louis, Missouri, United States, 63110