Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents

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Brief Title

Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents

Official Title

A Single Center Dose Ranging Pilot Study of (+)-Epicatechin in Non-ambulatory Adolescents With Duchenne Muscular Dystrophy and Pre-symptomatic Cardiac Dysfunction

Brief Summary

      This single center open-label pilot study will enroll 15 non-ambulatory children with
      Duchenne muscular dystrophy at least 8 years of age and who demonstrate pre-clinical
      cardiomyopathy (defined as a cardiac ejection fraction >55% with abnormal LV strain by
      cardiac MRI). They will receive (+)-epicatechin at one of three doses during an 8-week
      dose-ranging study with assessments at baseline, 2 Weeks, 4weeks, and 8 weeks. The study will
      determine optimal dosing for future cardiac efficacy studies based on serum / plasma
      biomarker response using follistatin: myostatin ratio, nitrite/nitrate ratio, cardiac
      troponins and cardiac BNP. Secondary endpoints will include additional biomarker assessments
      by SOMAscanTM, cardiac functional evaluations by cardiac MRI (LV strain), and echocardiogram
      (LV strain by speckle tracking) and measures of strength, range of motion and mobility, and
      clinical safety assessments. Results of secondary endpoint analysis will be used to refine
      design of subsequent clinical trials powered to detect changes in clinical outcomes.
    


Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Pharmacokinetics Outcome: Absolute Values of (+)-Epicatechin Serum Concentration, Pre-dose (Trough) and 2 Hours Post-dose (Peak)

Secondary Outcome

 Clinical Outcome: Percent of Normalized Upper Extremity Reachable Surface Area at Week 4 and Week 8

Condition

Duchenne Muscular Dystrophy

Intervention

(+)- Epicatechin

Study Arms / Comparison Groups

 Cohort 1
Description:  8-weeks open-label (+)- Epicatechin at 25mg/day twice per day,

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

15

Start Date

November 2016

Completion Date

July 2018

Primary Completion Date

July 2018

Eligibility Criteria

        Inclusion Criteria:

          -  Male

          -  Age 8 years to 17 years

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

          -  Weight 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.
      

Gender

Male

Ages

8 Years - 17 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02964377

Organization ID

951753


Responsible Party

Sponsor-Investigator

Study Sponsor

Craig McDonald, MD

Collaborators

 Cardero Therapeutics, Inc.

Study Sponsor

, , 


Verification Date

November 2021