(-)- Epicatechin Becker Muscular Dystrophy

NCT ID: NCT03236662

Last Updated: 2021-11-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 2 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-11-30
• Study Completion Date: 2017-12-31

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment

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

Group Type: EXPERIMENTAL

(-)-Epicatechin

Intervention Type: DRUG

Interventions

(-)-Epicatechin

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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).

Exclusion Criteria

• 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%

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Cardero Therapeutics, Inc.

INDUSTRY

Sponsor Role: collaborator

Craig McDonald, MD

OTHER

Sponsor Role: lead

Responsible Party

Craig McDonald, MD

Principal Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Locations

UC Davis Medical Center

Sacramento, California, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

767161

Identifier Type: -

Identifier Source: org_study_id