Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT04386304
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04386304
Recruitment Status : Active, not recruiting
First Posted : May 13, 2020
Last Update Posted : January 11, 2021
Sponsor:
Information provided by (Responsible Party):
Epirium Bio Inc.

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).

Condition or disease Intervention/treatment Phase
Becker Muscular Dystrophy Drug: (+)-Epicatechin Phase 1

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021


Arm Intervention/treatment
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.
Drug: (+)-Epicatechin
(+)-Epicatechin is a synthetic flavanol
Other Names:
  • EB 002
  • EPM-01




Primary Outcome Measures :
  1. 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).


Secondary Outcome Measures :
  1. Change in cardiac function as assessed by cardiac magnetic resonance imaging (MRI) [ Time Frame: Through study completion, up to 1 year ]
  2. 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 ]
  3. Change in muscle function as assessed by 6-minute walk test (6MWT) [ Time Frame: Through study completion, up to 1 year ]
  4. Change in muscle function as assessed by Time to Run/Walk 10-meter Test (TTRW10) [ Time Frame: Through study completion, up to 1 year ]
  5. Change in muscle function as assessed by Time to 4-stair Climb Test (TT4SC) [ Time Frame: Through study completion, up to 1 year ]
  6. Change in muscle function as assessed by Time to Run/Walk 100-meter Test (TTRW100) [ Time Frame: Through study completion, up to 1 year ]
  7. 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 ]
  8. Change in muscle biomarkers of regeneration in biopsy specimens (e.g. follistatin) [ Time Frame: Through study completion, up to 1 year ]
  9. Change in plasma biomarkers of muscle regeneration (e.g. follistatin, myostatin) [ Time Frame: Through study completion, up to 1 year ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   16 Years to 59 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

INCLUSION CRITERIA:

  1. Participant must be ≥16 to <60 years of age.
  2. Genotype confirmation showing a mutation of the dystrophin gene.
  3. Ambulation - participants must show a history of ambulation past the age of 16 years, with continued ambulation thereafter.
  4. 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:

  1. A diagnosis of other neurological diseases or presence of relevant somatic disorders that are not related to Becker muscular dystrophy.
  2. Participants with a history of migraine headaches requiring medical attention and active treatment within the past 6 months.
  3. Participants with allergies to chocolate or cocoa.
  4. 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.
  5. Presence of a concomitant neurologic disease (e.g., Parkinson's disease) that could negatively impact mobility or balance.
  6. Symptomatic heart failure (New York Heart Association Class III or IV) or known left ventricular ejection fraction <40% by echocardiogram.
  7. Presence of documented intrinsic lung disease (e.g., chronic obstructive pulmonary disease, pulmonary fibrosis).
  8. Evidence of current liver disease or impairment.
  9. Inadequate renal function.
  10. Platelet count, WBC count, and hemoglobin at Screening <Lower Limit of Normal (LLN).
  11. 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

Information from the National Library of Medicine

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


Locations
Layout table for location information
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
Sponsors and Collaborators
Epirium Bio Inc.
Investigators
Layout table for investigator information
Study Director: Chief Medical Officer Epirium Bio Inc.
Layout table for additonal information
Responsible Party: Epirium Bio Inc.
ClinicalTrials.gov Identifier: NCT04386304    
Other Study ID Numbers: EPM-01-101
First Posted: May 13, 2020    Key Record Dates
Last Update Posted: January 11, 2021
Last Verified: January 2021

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked