Overview

Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy

Status:
Active, not recruiting
Trial end date:
2021-12-01
Target enrollment:
0
Participant gender:
Male
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 Dystrophy (BMD).
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Epirium Bio Inc.
Criteria
INCLUSION CRITERIA:

1. Participant must be ≥16 to <60 years of age.

2. Genotype confirmation showing a mutation of the dystrophin gene.

3. Ambulation - participants must show a history of ambulation past the age of 16 years,
with continued ambulation thereafter.

4. 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:

1. A diagnosis of other neurological diseases or presence of relevant somatic disorders
that are not related to Becker muscular dystrophy.

2. Participants with a history of migraine headaches requiring medical attention and
active treatment within the past 6 months.

3. Participants with allergies to chocolate or cocoa.

4. 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.

5. Presence of a concomitant neurologic disease (e.g., Parkinson's disease) that could
negatively impact mobility or balance.

6. Symptomatic heart failure (New York Heart Association Class III or IV) or known left
ventricular ejection fraction <40% by echocardiogram.

7. Presence of documented intrinsic lung disease (e.g., chronic obstructive pulmonary
disease, pulmonary fibrosis).

8. Evidence of current liver disease or impairment.

9. Inadequate renal function.

10. Platelet count, WBC count, and hemoglobin at Screening
11. 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