(-)- Epicatechin Becker Muscular Dystrophy

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Becker Muscular Dystrophy	Drug: (-)-Epicatechin	Phase 2

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	10 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
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 Start Date :	November 2016
Estimated Primary Completion Date :	November 2017
Estimated Study Completion Date :	May 2018

Arms and Interventions

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Arm	Intervention/treatment
Experimental: Treatment; (-)-epicatechin 50mg twice per day (100mg per day total dose)	Drug: (-)-Epicatechin

Outcome Measures

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Primary Outcome Measures :

1. Peripheral venous blood will be collected to evaluate blood biomarkers [ Time Frame: 48 weeks ]
   Biomarkers: Plasma Follistatin, Plasma Myostatin, Plasma Follistatin: Plasma Myostatin ratio, Plasma Nitrates/ SNO, Plasma BNP, Plasma Creatine Kinase, Plasma MMP-9, Plasma TNF-Alpha, Plasma TGF-Beta

Secondary Outcome Measures :

1. Graded exercise test using a recumbent cycle ergometer [ Time Frame: baseline and at 2-minute intervals ]
   blood lactate measured
2. 6-minute walk test [ Time Frame: 48 weeks ]
   Measurements recorded will include 25-meter split times and total distance traveled.

Other Outcome Measures:

1. Exploratory Proteomics [ Time Frame: 48 weeks ]
   Collection of plasma samples for proteomics analysis.

Eligibility Criteria

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Ages Eligible for Study:	18 Years to 70 Years (Adult, Senior)
Sexes Eligible for Study:	Male
Accepts Healthy Volunteers:	No

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%

Contacts and Locations

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Contacts

Contact: Alina Nicorici, BS	916-734-0968	arnicorici@ucdavis.edu

Locations

United States, California
UC Davis Medical Center	Recruiting
Sacramento, California, United States, 95817
Contact: Alina Nicorici, BS 916-734-0968 arnicorici@ucdavis.edu
Principal Investigator: Craig M McDonald, MD
Sub-Investigator: Erik K Henricson, PhD, MPH

Sponsors and Collaborators

University of California, Davis

Cardero Therapeutics, Inc.

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	University of California, Davis
ClinicalTrials.gov Identifier:	NCT03236662 History of Changes
Other Study ID Numbers:	767161
First Posted:	August 2, 2017
Last Update Posted:	August 2, 2017
Last Verified:	July 2017

Keywords provided by University of California, Davis:

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

Additional relevant 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