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

  • STATUS
    Recruiting
  • participants needed
    20
  • sponsor
    Epirium Bio Inc.
Send
Updated on 16 February 2024
See if I qualify

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).

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.

Details
Condition Muscular Dystrophy, Muscular Dystrophy, DUCHENNE MUSCULAR DYSTROPHY
Age 16years - 59years
Treatment (+)-Epicatechin
Clinical Study IdentifierNCT04386304
SponsorEpirium Bio Inc.
Last Modified on16 February 2024

Eligibility

 Yes No Not Sure

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
Clear my responses
Read more

How to participate?

Step 1 Connect with a study center
Message sent successfully.
We have submitted the information you provided to the research team at the location you chose. For your records, we have sent a copy of the message to your email address.
If you would like to be informed of other studies that may be of interest to you, you may sign up for Patient Notification Service.
Sign up

Send a message

Enter your contact details to connect with study team

Email

View email

Phone

Phone country flag

View phone
Investigator Avatar

Primary Contact

First name*
Last name*
Email*
Phone number*
Other language

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.