Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02964377
• Date of registration: 07/11/2016
• Prospective Registration: No
• Primary sponsor: Craig McDonald, MD
• Public title: Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
• Scientific title: A Single Center Dose Ranging Pilot Study of (+)-Epicatechin in Non-ambulatory Adolescents With Duchenne Muscular Dystrophy and Pre-symptomatic Cardiac Dysfunction
• Date of first enrolment: November 2016
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02964377
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male

- Age 8 years to 17 years

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

- Weight
- 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.



Age minimum: 8 Years
Age maximum: 17 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: (+)- Epicatechin

Primary Outcome(s)

Pharmacokinetics Outcome: (+)-epicatechin peak (Cmax) serum concentration [Time Frame: Baseline, Week 2, Week 4, Week 8]

Pharmacokinetics Outcome: (+)-epicatechin trough (Cmin) serum concentration [Time Frame: Baseline, Week 2, Week 4, Week 8]

Safety: Clinical laboratory blood chemistry evaluation [Time Frame: Screening, Baseline, Week 2, Week 4, Week 8]

Clinical Outcome: Percent change in cardiac ejection fraction and shortening fraction by MRI [Time Frame: Change from Baseline to Week 4 and Week 8]

Laboratory Outcome: Change in plasma follistatin:myostatin ratio [Time Frame: Change from Baseline to Week 4 and Week 8]

Safety: Complete blood count evaluation [Time Frame: Screening, Baseline, Week 2, Week 4, Week 8]

Safety: Urinalysis [Time Frame: Screening, Baseline, Week 2, Week 4, Week 8]

Secondary Outcome(s)

Person-Reported Outcome: Percent change in Person-Reported Outcome Measure Upper Limb (PROM-UL) functional capacity score. [Time Frame: Change from Baseline to Week 4 and Week 8]

Clinical Outcome: Percent change in 6-minute cycle test maximal attained revolutions. [Time Frame: Change from Baseline to Week 4 and Week 8]

Clinical Outcome: Percent change in normalized upper extremity reachable surface area. [Time Frame: Change from Baseline to Week 4 and Week 8]

Clinical Outcome: Percent change in Performance of the Upper Limb Assessment score. [Time Frame: Change from Baseline to Week 4 and Week 8]

Person-Reported Outcome: Percent change in POSNA Pediatric Outcomes Data Collection Instrument (PODCI) quality of life instrument score [Time Frame: Change from Baseline to Week 4 and Week 8]

Secondary ID(s)

951753

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cardero Therapeutics, Inc.