A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy — HOPE-3  

Nervous System Diseases Clinical Trial

Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

Status Recruiting

Clinical Trial Summary

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.

Clinical Trial Description

Up to 102 eligible study subjects will participate in this two cohort study. Cohort A will enroll approximately 58 subjects randomized in a 1:1 ratio (active:placebo). Subjects randomized to the active treatment group in Cohort A will receive CAP-1002A (manufactured at Capricor's manufacturing facility in Los Angeles, CA). Once Cohort A enrollment is completed, Cohort B enrollment will begin. Cohort B will enroll approximately 44 participants randomized in a 1:1 ratio (active:placebo). Subjects randomized to the active treatment group in Cohort B will receive CAP-1002B (manufactured at Capricor's manufacturing facility in San Diego, CA). Both cohorts will include visits at Screening, Baseline/Day 1, Month 1, and Months 3, 6, 9, and 12. Subjects will receive IV infusions of CAP-1002 or placebo on Day 1 and Months 3, 6, and 9. All subjects will then be eligible to receive additional IV infusions of CAP-1002 at Month 12, 15, 18, and 21 as part of the open-label phase of the study.

A primary analysis of efficacy and safety will be performed for each individual cohort at Month 12 following 4 administrations of CAP-1002 or placebo.

The primary efficacy endpoint is the mean change from baseline in upper limb function as assessed by Performance of the Upper Limb test, version 2.0 [PUL 2.0] Total Score at the 12-month time point. Secondary endpoints evaluated at the 12-month time point include assessment of changes in cardiac muscle function and structure by cardiac magnetic resonance imaging [cMRI], changes in hand-to-mouth function [eat 10-bites assessed by the Duchenne Video Assessment (DVA)], quality of life assessments, and biomarker analysis for creatine kinase MB isoenzyme (CK-MB).

Safety evaluations will include adverse events, concomitant medications, physical exam, vital signs, and clinical laboratory testing.

Following the initial 4 infusions in Cohort A or Cohort B, all subjects will be eligible to participate in a 12-month open label extended assessment period and receive up to 4 additional IV infusions of CAP-1002 once every 3 months. An analysis of extended safety and efficacy will be conducted after all subjects have completed the Month 24 visit. ;

Related Conditions & MeSH terms

• Genetic Diseases, Inborn
• Genetic Diseases, X-Linked
• Muscular Diseases
• Muscular Disorders, Atrophic
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne
• Nervous System Diseases
• Neuromuscular Diseases

• NCT number: NCT05126758
• Study type: Interventional
• Source: Capricor Inc.
• Contact: Kevin Berth
• Phone: 858-727-1755
• Email: clinicaltrialsgov@capricor.com
• Status: Recruiting
• Phase: Phase 3
• Start date: June 22, 2022
• Completion date: December 2026