A Phase 1, Open-label, Dose Escalation Study to Evaluate the Safety and Preliminary Efficacy of Orally Administered (+)-Epicatechin in Patients With Becker or Becker-like Muscular Dystrophy With Continued Ambulation Past 16 Years of Age
Overview
- Phase
- Phase 1
- Intervention
- (+)-Epicatechin
- Conditions
- Becker Muscular Dystrophy
- Sponsor
- Epirium Bio Inc.
- Enrollment
- 22
- Locations
- 3
- Primary Endpoint
- Number of participants with treatment-emergent adverse events (TEAEs)
- Status
- Completed
- Last Updated
- 4 years ago
Overview
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 Dystrophy (BMD).
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, cardiac imaging, and on clinical function assessments of participants' muscle strength. All patients will receive oral (+)-epicatechin for a total duration of approximately 52 weeks. Three doses of (+)-epicatechin will be tested in sequential 2 month periods with total daily doses of 75, 150, and 225 mg/day (+)-epicatechin. Doses will be escalated every 2 months, if tolerated, for the first 6 months of the study. Participants will then continue to receive the highest does they tolerated for an additional 6 months.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Dose escalation of (+)-epicatechin
Subjects will receive escalating doses of (+)-epicatechin starting at 75 mg/day and progressing to 150 mg/day and 225 mg/day with 2 months treatment duration for each dose. Subjects will continue treatment on the individual's maximum tolerated dose for another 6 months.
Intervention: (+)-Epicatechin
Outcomes
Primary Outcomes
Number of participants with treatment-emergent adverse events (TEAEs)
Time Frame: Through study completion, up to 1 year
The TEAEs will be graded using the adult National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0).
Secondary Outcomes
- Change in cardiac function as assessed by cardiac magnetic resonance imaging (MRI)(Through study completion, up to 1 year)
- Change in cardiac function as assessed by plasma biomarkers [e.g. pro-B-type natriuretic peptide (pro-BNP), nitrates].(Through study completion, up to 1 year)
- Change in muscle function as assessed by 6-minute walk test (6MWT)(Through study completion, up to 1 year)
- Change in muscle function as assessed by Time to Run/Walk 10-meter Test (TTRW10)(Through study completion, up to 1 year)
- Change in muscle function as assessed by Time to 4-stair Climb Test (TT4SC)(Through study completion, up to 1 year)
- Change in muscle function as assessed by Time to Run/Walk 100-meter Test (TTRW100)(Through study completion, up to 1 year)
- Change in muscle structure and function as assessed by Western blot analysis of biopsy specimens (e.g. dystrophin expression)(Through study completion, up to 1 year)
- Change in muscle biomarkers of regeneration in biopsy specimens (e.g. follistatin)(Through study completion, up to 1 year)
- Change in plasma biomarkers of muscle regeneration (e.g. follistatin, myostatin)(Through study completion, up to 1 year)