(-)- Epicatechin Becker Muscular Dystrophy

NCT03236662 | Completed Phase 2 Results

• 1 other identifier: 767161
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial. This single center study will enroll up to 10 adults who will receive the purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks. After screening visits, participants will be enrolled in the study if they meet all inclusion criteria. They will be evaluated at screening, baseline, and weeks 4, 8, 12, 24, 16 and 48. The main criterion for success of the study will be presence of one or more biologic or strength and performance outcome measures that yield a response magnitude that allows for sufficient power in a Phase II B study with a sample size of 30 individuals.

Conditions

Becker Muscular Dystrophy

Keywords

BMD; Becker muscular dystrophy; epicatechin; clinical trial; neuromuscular disease

Outcome Measures

Primary Outcomes (9)

• Plasma Follistatin

  blood biomarker concentration

  48 weeks

• Plasma Myostatin

  blood biomarker concentration

  48 weeks

• Plasma Nitrates/ SNO

  blood biomarker concentration

  48 weeks

• Plasma BNP

  blood biomarker concentration

  48 weeks

• Plasma Creatine Kinase

  blood biomarker concentration

  48 weeks

• Plasma MMP-9

  blood biomarker concentration

  48 weeks

• Plasma TNF-Alpha

  blood biomarker concentration

  48 weeks

• Plasma TGF-Beta

  blood biomarker concentration

  48 weeks

• Plasma Follistatin:Myostain Ratio

  Ratio of plasma follistatin to plasma myostatin

  48 weeks

Secondary Outcomes (2)

• Graded Exercise Test Using a Recumbent Cycle Ergometer

  baseline and at 2-minute intervals

• 6-minute Walk Test

  48 weeks

Other Outcomes (1)

• Exploratory Proteomics

  48 weeks

Study Arms (1)

Treatment

EXPERIMENTAL

(-)-epicatechin 50mg twice per day (100mg per day total dose)

Drug: (-)-Epicatechin

Interventions

(-)-Epicatechin DRUG

Treatment

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Prior participation in UCD0113 BMD epicatechin pilot study
• Male
• Age 18 years to 70 years
• Average to low daily physical activity
• Ability to ambulate for 75 meters without assistive devices
• Diagnosis of BMD confirmed by at least one the following:
• Dystrophin immunofluorescence and/or immunoblot showing partial dystrophin deficiency, and clinical picture consistent with typical BMD, or
• Gene deletions test positive (missing one or more exons) of the dystrophin gene, where reading frame can be predicted as 'in-frame', and clinical picture consistent with typical BMD, or
• Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) that can be definitely associated with BMD, with a typical clinical picture of BMD, or
• Positive family history of BMD confirmed by one of the criteria listed above in a sibling or maternal uncle, and clinical picture typical of BMD.
• Hematology profile within normal range
• Baseline laboratory safety chemistry profile within normal range
• No plan to change exercise regimen during study participation
• Nutritional, herbal and antioxidant supplements taken with the intent of maintaining or improving skeletal muscle strength or functional mobility have been discontinued at least 2 weeks prior to screening (daily multivitamin use is acceptable).

You may not qualify if:

• Currently enrolled in another treatment clinical trial.
• History of significant concomitant illness or significant impairment of renal or hepatic function.
• Use of regular daily aspirin or other medication with antiplatelet effects within 3 weeks of first dose of study medication.
• Regular participation in vigorous exercise.
• Symptomatic heart failure with cardiac ejection fraction \<25%

Sponsors & Collaborators

• Craig McDonald, MD: lead
• Cardero Therapeutics, Inc.: collaborator

Study Sites (1)

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Neuromuscular Diseases

Interventions

Catechin

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal 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

Limitations and Caveats

Small number of subjects analyzed.

Results Point of Contact

• Title: Dr. Craig McDonald, Professor and Chair
• Organization: UC Davis Health

cmmcdonald@ucdavis.edu

(916) 734-4293

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: November 7, 2016
• First Posted: August 2, 2017
• Study Start: November 1, 2016
• Primary Completion: November 1, 2017
• Study Completion: December 1, 2017
• Last Updated: November 24, 2021
• Results First Posted: July 26, 2021

Record last verified: 2021-11

Locations

US (1)