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

NCT04386304 | Completed Phase 1 FDA Drug

• 1 other identifier: EPM-01-101
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 3

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).

Conditions

Becker Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• 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).

  Through study completion, up to 1 year

Secondary Outcomes (9)

• 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

Study Arms (1)

Dose escalation of (+)-epicatechin

EXPERIMENTAL

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.

Drug: (+)-Epicatechin

Interventions

(+)-Epicatechin DRUG

(+)-Epicatechin is a synthetic flavanol

Also known as: EB 002, EPM-01

Dose escalation of (+)-epicatechin

Eligibility Criteria

• Age: 16 Years - 59 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Epirium Bio Inc.: lead

Study Sites (3)

UCLA Dept of Human Genetics

Los Angeles, California, 90095, United States

Location

University of California - Davis Department of Physical Medicine and Rehabilitation

Sacramento, California, 95817, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Catechin

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Chromans; Benzopyrans; Pyrans; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Flavonoids; Chromones; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Chief Medical Officer

  Epirium Bio Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 1, 2020
• First Posted: May 13, 2020
• Study Start: July 13, 2020
• Primary Completion: March 1, 2022
• Study Completion: March 1, 2022
• Last Updated: March 23, 2022

Record last verified: 2022-03

Locations

US (3)