Study of nutritonal extract in treatment of non- ambulatory children and teens with Duchenne Muscular Dystrophy

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"Experimental medicine, (+)-epicatechin, (nutritional extract, purified from tea,also found in cocoa) to treat Duchenne Muscular Dystrophy"
Age:
8 to 17 years old
Gender:
Male
Healthy Volunteers:
No
Keywords:
duchenne muscular dystrophy
Type:
Drug study, Phase I/II
Investigator:
Description
This study will look at an experimental medicine called (+)- Epicatechin. (+)-Epicatechin is purified from tea as a nutritional extract and is also found in cocoa. It has been shown to improve muscle structure and function in previous studies of animals and humans. (+)-Epicatechin will be evaluated for the treatment of non-ambulatory children and teens with Duchenne muscular dystrophy (DMD). The main objective of this study is to evaluate the effect and safety of 3 different doses of epicatechin over ashort period of time in 15 people with DMD. The study will also look at changes in biomarkers (small molecules in the blood) that will determine whether the study drug is affecting the function of the heart
and skeletal muscles. If the results are positive, researchers will conduct a longer and larger study to evaluate whether the study drug can make people feel better.
This study requires
Medical History
Physical/Neurological Examination
Review of Medications
Urinalysis
Blood draw
Electrocardiogram
Cardiac function testing
Strength, Range of motion, mobility testing
Body compositon testing
Study medication
Medication diary
Who can participate?
Inclusion Criteria:

- Male

- Age 8 years to 17 years

- Non-Ambulatory (unable to complete 10m run/walk under 10s)

- Weight </=100Kg

- Diagnosis of DMD confirmed by at least one the following:

- Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, or

- Gene deletions test positive (missing one or more exons) of the dystrophin gene, where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with typical DMD, 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 DMD, with a typical clinical picture of DMD, or

- Positive family history of DMD confirmed by one of the criteria listed above in a sibling or maternal uncle, and clinical picture typical of DMD.

- Cardiac ejection fraction >55% on echocardiogram

- Use of nutritional, herbal and antioxidant supplements taken with the intent of maintaining or improving skeletal muscle strength or functional mobility has been discontinued at least 4 weeks prior to screening (daily multivitamin use is acceptable).

- Glucocorticoid therapy, if used, must have a stable weight-based dose for at least 3 months prior to enrollment

- Cardiac therapy, if used, includes prophylactic ACE inhibitors, aldosterone receptor antagonists (e.g.

spironolactone, eplerenone, etc.), and/or beta-blocker therapy, and must be stable for 3 months prior to enrollment.

- Hematology profile within normal range.

- Baseline laboratory safety chemistry profile within typical range for DMD (elevated ALT / AST acceptable in the absence of elevated GGT, elevated CK acceptable).


Exclusion Criteria:

- Inability to complete cardiac or strength, range of motion and mobility assessments per protocol

- Current enrollment 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.

- Cardiac symptoms that, in the opinion of the investigator, may be suggestive of imminent moderate to severe cardiac events, irrespective of LVEF.
Resources
Schedule
Study duration and period
Five visits over 12 weeks Each visit approximately 3 hours long
Recruitment period
From Oct. 4, 2016
Location
UC Davis Children's Hospital
2315 Stockton Boulevard
Sacramento, CA 95817
Contact
Laura Andrea Pulido-Ramirez
Research Topic
Conditions:
  • Duchenne Muscular Dystrophy

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