Grant Award Details

Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy
Grant Number: 
CLIN2-08334
Project Objective: 
  • To complete a Phase II clinical trial in Duchenne Muscular Dystrophy patients with cardiomyopathy, in which allogeneic Cardiosphere Derived Cells (CDCs) [aka CAP-1002] are infused one time via multi-vessel intracoronary delivery.

Investigator: 
Institution: 
Type: 
PI
Disease Focus: 
Heart Disease
Heart disease associated with Duchenne muscular dystrophy
Human Stem Cell Use: 
Adult Stem Cell
Award Value: 
$3,376,259
Status: 
Active

Progress Reports

Reporting Period: 
Operational Milestone #1

Grant Application Details

Application Title: 
  • Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy
Public Abstract: 

Therapeutic Candidate or Device

Allogeneic Cardiosphere-Derived Cells (CDCs, CAP-1002)

Indication

Duchenne Muscular Dystrophy Cardiomyopathy

Therapeutic Mechanism

Secretion of identified bioactive elements will reduce myocardial fibrosis and improve cardiac function.

Unmet Medical Need

Heart failure is the leading cause of death among young men with Duchenne Muscular Dystrophy. No specific therapies exist to treat this element of the disease. CAP-1002 is intended to treat the cardiomyopathy associated with DMD.

Project Objective

Phase 2 study completed

Major Proposed Activities

  • manufacture CAP-1002 in quantities sufficient to treat all subjects enrolled in the trial
  • enroll and treat all subjects per the clinical protocol
  • enroll and treat all subjects per the clinical protocol
Statement of Benefit to California: 

The applicant is a California-based organization. At least one clinical site within California will participate in the study.