Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy
Grant Award Details
Grant Type:
Grant Number:
CLIN2-08334
Investigator(s):
Human Stem Cell Use:
Award Value:
$3,376,259
Status:
Closed
Progress Reports
Reporting Period:
Operational Milestone #1
Reporting Period:
Final Operational Milestone #5
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
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.
Publications
- Neurology (2019): Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial. (PubMed: 30674601)
