Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT04386304|
Recruitment Status : Completed
First Posted : May 13, 2020
Last Update Posted : March 23, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Becker Muscular Dystrophy||Drug: (+)-Epicatechin||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||22 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||July 13, 2020|
|Actual Primary Completion Date :||March 1, 2022|
|Actual Study Completion Date :||March 1, 2022|
Experimental: 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.
(+)-Epicatechin is a synthetic flavanol
- 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).
- 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 ]
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|Ages Eligible for Study:||16 Years to 59 Years (Child, Adult)|
|Sexes Eligible for Study:||Male|
|Accepts Healthy Volunteers:||No|
- 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.
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04386304
|United States, California|
|UCLA Dept of Human Genetics|
|Los Angeles, California, United States, 90095|
|University of California - Davis Department of Physical Medicine and Rehabilitation|
|Sacramento, California, United States, 95817|
|United States, Missouri|
|Washington University School of Medicine|
|Saint Louis, Missouri, United States, 63110|
|Study Director:||Chief Medical Officer||Epirium Bio Inc.|
|Responsible Party:||Epirium Bio Inc.|
|Other Study ID Numbers:||
|First Posted:||May 13, 2020 Key Record Dates|
|Last Update Posted:||March 23, 2022|
|Last Verified:||March 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked