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Follistatin 315

Activin-Binding Protein, FSH-Suppressing Protein, FST-315, FS-315

Quick Stats
Studies 14
Trials 93
Completed PHASE1, PHASE2 INTERVENTIONAL NCT02964377

Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents

View on ClinicalTrials.gov Updated Dec 15, 2025

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.

Interventions

Name: (+)- Epicatechin
Type: DRUG
Description:

Primary Outcomes

Measure: Pharmacokinetics Outcome: Absolute Values of (+)-Epicatechin Serum Concentration, Pre-dose (Trough) and 2 Hours Post-dose (Peak)
TimeFrame: Pre-dose and 2 hours post-dose at baseline
Description: Pharmacokinetic evaluation for dose-response evaluation.
Measure: Pharmacokinetics Outcome: Absolute Values of (+)-Epicatechin Serum Concentration, Pre-dose (Trough) and 2 Hours Post-dose (Peak)
TimeFrame: Week 4
Description: Pharmacokinetic evaluation for dose-response evaluation.
Measure: Laboratory Outcome: Absolute Plasma Follistatin:Myostatin Ratio at Baseline, Week 4 and Week 8
TimeFrame: Baseline, Week 4 and Week 8
Description: Evaluation of follistatin:myostatin ratio from plasma samples.
Measure: Clinical Outcome: Mean Percent of Baseline Cardiac Ejection Fraction by MRI
TimeFrame: Week 8
Description: Evaluation of change in cardiac volume and performance, as measured by the mean percent of baseline ejection fraction using Cardiac MRI, measured at 8 weeks.
Measure: Safety: Number of Participants Who Experienced Treatment-Related Laboratory Abnormalities
TimeFrame: Study duration (8 weeks)
Description: Treatment-related laboratory abnormalities, defined as values outside of the typical range for Duchenne Muscular Dystrophy. Safety laboratory tests included blood chemistry panel, complete blood count w/ differential panel, \& urinalysis assessments for clinical safety monitoring.
Measure: Laboratory Outcome: Absolute Values of Nitric Oxide (AU) Measured by ELISA
TimeFrame: Baseline, Week 4, Week 8
Description: Proteomics evaluation of plasma biomarkers to confirm intervention-responsive pathophysiological pathways, using enzyme-linked immunosorbent assay (ELISA).
Measure: Laboratory Outcome: Absolute Values of Carbonylation (AU) Measured by ELISA
TimeFrame: Baseline, Week 4, Week 8
Description: Proteomics evaluation of plasma biomarkers to confirm intervention-responsive pathophysiological pathways, using enzyme-linked immunosorbent assay (ELISA).
Measure: Laboratory Outcome: Absolute Values of Follistatin (AU) Measured by ELISA
TimeFrame: Baseline, Week 4, Week 8
Description: Proteomics evaluation of plasma biomarkers to confirm intervention-responsive pathophysiological pathways, using enzyme-linked immunosorbent assay (ELISA).
Measure: Laboratory Outcome: Absolute Values of Myostatin (AU) Measured by ELISA
TimeFrame: Baseline, Week 4, Week 8
Description: Proteomics evaluation of plasma biomarkers to confirm intervention-responsive pathophysiological pathways, using enzyme-linked immunosorbent assay (ELISA).

Trial Information

NCT ID

NCT02964377

Status

Completed

Study Type

INTERVENTIONAL

Phases

PHASE1, PHASE2

Sponsor

Craig McDonald, MD

Last Updated

December 15, 2025

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