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

NCT03406780 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2025-05-28

Study results available
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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.

Conditions

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

Interventions

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

Sponsors & Collaborators

  • Capricor Inc.

    lead INDUSTRY

Principal Investigators

  • Craig McDonald, MD · University of California, Davis

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
10 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-04-04
Primary Completion
2020-03-10
Completion
2020-03-10
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03406780 on ClinicalTrials.gov