Back to results(-)- Epicatechin Becker Muscular Dystrophy
Primary Purpose
Becker Muscular Dystrophy
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
(-)-Epicatechin
Sponsored by
About this trial
This is an interventional treatment trial for Becker Muscular Dystrophy focused on measuring BMD, Becker muscular dystrophy, epicatechin, clinical trial, neuromuscular disease
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%
Sites / Locations
- UC Davis Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treatment
Arm Description
(-)-epicatechin 50mg twice per day (100mg per day total dose)
Outcomes
Primary Outcome Measures
Plasma Follistatin
blood biomarker concentration
Plasma Myostatin
blood biomarker concentration
Plasma Nitrates/ SNO
blood biomarker concentration
Plasma BNP
blood biomarker concentration
Plasma Creatine Kinase
blood biomarker concentration
Plasma MMP-9
blood biomarker concentration
Plasma TNF-Alpha
blood biomarker concentration
Plasma TGF-Beta
blood biomarker concentration
Plasma Follistatin:Myostain Ratio
Ratio of plasma follistatin to plasma myostatin
Secondary Outcome Measures
Graded Exercise Test Using a Recumbent Cycle Ergometer
blood lactate measured
6-minute Walk Test
Measurements recorded will include 25-meter split times and total distance traveled.
Full Information
NCT ID
NCT03236662
First Posted
November 7, 2016
Last Updated
November 22, 2021
Sponsor
Craig McDonald, MD
Collaborators
Cardero Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03236662
Brief Title
(-)- Epicatechin Becker Muscular Dystrophy
Official Title
UCD0115B: An Open-label Extension Study of Purified Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
November 2021
Overall Recruitment Status
Completed
Study Start Date
November 2016 (Actual)
Primary Completion Date
November 2017 (Actual)
Study Completion Date
December 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Craig McDonald, MD
Collaborators
Cardero Therapeutics, Inc.
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Becker Muscular Dystrophy
Keywords
BMD, Becker muscular dystrophy, epicatechin, clinical trial, neuromuscular disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Treatment
Arm Type
Experimental
Arm Description
(-)-epicatechin 50mg twice per day (100mg per day total dose)
Intervention Type
Drug
Intervention Name(s)
(-)-Epicatechin
Primary Outcome Measure Information:
Title
Plasma Follistatin
Description
blood biomarker concentration
Time Frame
48 weeks
Title
Plasma Myostatin
Description
blood biomarker concentration
Time Frame
48 weeks
Title
Plasma Nitrates/ SNO
Description
blood biomarker concentration
Time Frame
48 weeks
Title
Plasma BNP
Description
blood biomarker concentration
Time Frame
48 weeks
Title
Plasma Creatine Kinase
Description
blood biomarker concentration
Time Frame
48 weeks
Title
Plasma MMP-9
Description
blood biomarker concentration
Time Frame
48 weeks
Title
Plasma TNF-Alpha
Description
blood biomarker concentration
Time Frame
48 weeks
Title
Plasma TGF-Beta
Description
blood biomarker concentration
Time Frame
48 weeks
Title
Plasma Follistatin:Myostain Ratio
Description
Ratio of plasma follistatin to plasma myostatin
Time Frame
48 weeks
Secondary Outcome Measure Information:
Title
Graded Exercise Test Using a Recumbent Cycle Ergometer
Description
blood lactate measured
Time Frame
baseline and at 2-minute intervals
Title
6-minute Walk Test
Description
Measurements recorded will include 25-meter split times and total distance traveled.
Time Frame
48 weeks
Other Pre-specified Outcome Measures:
Title
Exploratory Proteomics
Description
Collection of plasma samples for proteomics analysis.
Time Frame
48 weeks
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
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%
Facility Information:
Facility Name
UC Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
12. IPD Sharing Statement
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(-)- Epicatechin Becker Muscular Dystrophy
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