NCT03406780

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

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Capricor Inc. · Industry
• Sex: Male
• Age: 10 Years
• Healthy volunteers: Not accepted

Summary

HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne Muscular Dystrophy (DMD). 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 a 12-month period.

Detailed description

* Approximately 20 eligible study participants will be randomized to either CAP-1002 or placebo in a 1:1 ratio. * The trial will include visits at Screening, Baseline/Day 1, Week 4, and Months 3, 6, 9, and 12 with IV infusions of CAP-1002 or placebo on Day 1 and Months 3, 6, and 9. * Safety evaluations will include adverse events, concomitant medications, physical exam, vital signs, 12-lead ECG, and clinical laboratory testing. * Efficacy will be evaluated by Performance of the Upper Limb, cardiac MRI, pulmonary function testing, North Star Ambulatory Assessment (ambulatory subjects only), strength testing, and quality of life. * If trial data suggests an appropriate risk/benefit profile of CAP-1002, Capricor, upon the recommendation of the Data Safety Monitoring Board (DSMB), will introduce an open-label extension study to offer CAP-1002 to study participants who were randomized to placebo and completed all trial visits during the 12-month period.

Conditions

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

Interventions

Type	Name	Description
BIOLOGICAL	CAP-1002	The active pharmaceutical ingredient in CAP-1002 is Cardiosphere-Derived Cells (CDCs). CDCs are known to secrete numerous bioactive elements (growth factors, exosomes) which impact the therapeutic benefits of the cell-based therapy. The mechanism of action is the composite ability to be immunomodulatory, anti-fibrotic, anti-inflammatory, and pro-angiogenic.
DRUG	Placebo	Placebo

Timeline

• Start date: 2018-04-04
• Primary completion: 2020-03-10
• Completion: 2020-03-10
• First posted: 2018-01-23
• Last updated: 2025-05-28
• Results posted: 2025-02-24

Locations

9 sites across 1 country: United States

Regulatory

• FDA-regulated drug study