Becker Muscular DystrophyPhase 1NCT04386304
Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy
A Phase 1 study testing (+)-Epicatechin in people with Becker Muscular Dystrophy, run by Epirium Bio Inc. at 3 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 16 to 59 years, men only
- Conditions the study is for
- Becker Muscular Dystrophy
- What is being tested?
- (+)-Epicatechin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 3 sitesUnited States
- Study dates
- 13 Jul 2020to 1 Mar 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 59 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
UCLA Dept of Human Genetics
Los Angeles, California, 90095
University of California - Davis Department of Physical Medicine and Rehabilitation
Sacramento, California, 95817
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
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
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).
Read the 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.
- Conditions
- Becker Muscular Dystrophy
- Being tested
- (+)-Epicatechin
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
(+)-EpicatechinDrug
Also called EB 002, EPM-01
(+)-Epicatechin is a synthetic flavanol
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: (+)-Epicatechin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Through study completion, up to 1 year
Secondary outcomes (9)
Change in cardiac function as assessed by cardiac magnetic resonance imaging (MRI)
Measured: 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].
Measured: Through study completion, up to 1 year
Change in muscle function as assessed by 6-minute walk test (6MWT)
Measured: Through study completion, up to 1 year
Change in muscle function as assessed by Time to Run/Walk 10-meter Test (TTRW10)
Measured: Through study completion, up to 1 year
Change in muscle function as assessed by Time to 4-stair Climb Test (TT4SC)
Measured: Through study completion, up to 1 year
Change in muscle function as assessed by Time to Run/Walk 100-meter Test (TTRW100)
Measured: 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)
Measured: Through study completion, up to 1 year
Change in muscle biomarkers of regeneration in biopsy specimens (e.g. follistatin)
Measured: Through study completion, up to 1 year
Change in plasma biomarkers of muscle regeneration (e.g. follistatin, myostatin)
Measured: Through study completion, up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2020
- Study start13 Jul 2020
- Primary completion1 Mar 2022
- Study completion1 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Becker Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Epirium Bio Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chief Medical Officer, Study director, Epirium Bio Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04386304
- Sponsor's protocol ID
- EPM-01-101
- Status last verified
- Mar 2022
- First submitted
- 1 May 2020
- Last updated
- 23 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04386304
- Acronym / sponsor ID
- EPM-01-101
- Conditions
- Becker Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Epirium Bio Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Jul 2020 (actual)
- Primary completion
- 1 Mar 2022 (actual)
- Study completion
- 1 Mar 2022 (actual)
- First posted
- 13 May 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose escalation of (+)-epicatechin | Experimental | Drug: (+)-EpicatechinSubjects 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. |
Who may qualify
- Ages
- 16 to 59 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| (+)-EpicatechinAlso: EB 002, EPM-01(+)-Epicatechin is a synthetic flavanol | Drug | Dose escalation of (+)-epicatechin |
Primary outcome
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).
9 secondary outcomes
Change in cardiac function as assessed by cardiac magnetic resonance imaging (MRI)
Time frame: 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].
Time frame: Through study completion, up to 1 year
Change in muscle function as assessed by 6-minute walk test (6MWT)
Time frame: Through study completion, up to 1 year
Change in muscle function as assessed by Time to Run/Walk 10-meter Test (TTRW10)
Time frame: Through study completion, up to 1 year
Change in muscle function as assessed by Time to 4-stair Climb Test (TT4SC)
Time frame: Through study completion, up to 1 year
Change in muscle function as assessed by Time to Run/Walk 100-meter Test (TTRW100)
Time frame: 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)
Time frame: Through study completion, up to 1 year
Change in muscle biomarkers of regeneration in biopsy specimens (e.g. follistatin)
Time frame: Through study completion, up to 1 year
Change in plasma biomarkers of muscle regeneration (e.g. follistatin, myostatin)
Time frame: Through study completion, up to 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chief Medical Officer study director
Epirium Bio Inc.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing (+)-Epicatechin in people with Becker Muscular Dystrophy, run by Epirium Bio Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-emergent adverse events (TEAEs) (time frame: Through study completion, up to 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2022.