Back to results

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

Primary Purpose

Becker Muscular Dystrophy

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

(+)-Epicatechin

About this trial

This is an interventional treatment trial for Becker Muscular Dystrophy

Eligibility Criteria

16 Years - 59 Years (Child, Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

UCLA Dept of Human Genetics
University of California - Davis Department of Physical Medicine and Rehabilitation
Washington University School of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Dose escalation of (+)-epicatechin

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of participants with treatment-emergent adverse events (TEAEs)

The TEAEs will be graded using the adult National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0).

Secondary Outcome Measures

Change in cardiac function as assessed by cardiac magnetic resonance imaging (MRI)

Change in cardiac function as assessed by plasma biomarkers [e.g. pro-B-type natriuretic peptide (pro-BNP), nitrates].

Change in muscle function as assessed by 6-minute walk test (6MWT)

Change in muscle function as assessed by Time to Run/Walk 10-meter Test (TTRW10)

Change in muscle function as assessed by Time to 4-stair Climb Test (TT4SC)

Change in muscle function as assessed by Time to Run/Walk 100-meter Test (TTRW100)

Change in muscle structure and function as assessed by Western blot analysis of biopsy specimens (e.g. dystrophin expression)

Change in muscle biomarkers of regeneration in biopsy specimens (e.g. follistatin)

Change in plasma biomarkers of muscle regeneration (e.g. follistatin, myostatin)

Full Information

NCT ID

NCT04386304

First Posted

May 1, 2020

Last Updated

March 22, 2022

Sponsor

Epirium Bio Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04386304

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2022

Overall Recruitment Status

Completed

Study Start Date

July 13, 2020 (Actual)

Primary Completion Date

March 1, 2022 (Actual)

Study Completion Date

March 1, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Epirium Bio Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Becker Muscular Dystrophy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Sequential Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

22 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Dose escalation of (+)-epicatechin

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

(+)-Epicatechin

Other Intervention Name(s)

EB 002, EPM-01

Intervention Description

(+)-Epicatechin is a synthetic flavanol

Primary Outcome Measure Information:

Title

Number of participants with treatment-emergent adverse events (TEAEs)

Description

The TEAEs will be graded using the adult National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0).

Time Frame