The prospective, randomized Intracoronary CArdiosphere-Derived aUtologous StemCElls to reverse ventricUlar dySfunction (CADUCEUS) Phase I trial was a proof-of-concept study that evaluated the safety and efficacy of Capricor’s investigational, autologous cardiosphere-derived cell (CDC) product, CAP-1001.

The trial was conducted at Cedars-Sinai Medical Center and Johns Hopkins University and was sponsored by Cedars-Sinai Medical Center under the leadership of Eduardo Marbán, M.D., Ph.D., Director of the Cedars-Sinai Heart Institute and Co-Founder and Scientific Advisory Board Chairman of Capricor.

Status of Study:

Completed. Not enrolling.

Study Design:

The study enrolled patients 2-4 weeks after myocardial infarction at two U.S. sites, Cedars-Sinai Medical Center and Johns Hopkins University. An independent data coordinating center randomly allocated patients in a 2:1 ratio to receive CDCs or standard of care.

For patients assigned to receive CDCs, autologous cells were grown from endomyocardial biopsy specimens received from the same patient and were then infused into the infarct-related artery 1.5-3 months after myocardial infarction. Patients assigned to the control group were given routine care while undergoing all protocol-specified safety and efficacy assessments.

Efficacy in patients was assessed in terms of New York Heart Association class, the Minnesota Living with Heart Failure Questionnaire, 6-minute walk tests, and magnetic resonance imaging (MRI). Contrast-enhanced MRI studies were conducted at baseline, 6 months, and 12 months to assess the longevity of the treatment effects. MRI assessments measured scar mass and viable myocardial mass in the left ventricle, scar size, cardiac volumes, global function, and regional function in all patients.

Patient Population:

A total of 31 patients 18 years or older with a recent myocardial infarction (≤4 weeks) were enrolled in the trial. These patients had documented left ventricular dysfunction with ejection fraction (EF) between 25-45%.

Study Outcomes:

While CADUCEUS was designed to affirm the safety of taking a myocardial biopsy to generate the CDCs and infuse them into the patient, preliminary data showed that CDC therapy significantly reduced infarct (scar) size and increased healthy heart tissue. Findings showed that intracoronary infusion of autologous CDCs (CAP-1001) after myocardial infarction is safe, warranting the expansion of such therapy to a Phase II study.

The study challenged the long-standing convention that cardiac scarring is permanent and healthy heart muscle cannot be restored. CADUCEUS provided early evidence for therapeutic regeneration. The scars caused by the myocardial infarction shrank over the course of 12 months in patients treated with CAP-1001. Scientists observed a significant correlation between scar shrinkage and viability increases. Patients treated with CAP-1001 also demonstrated improved regional function and trends pointing toward the reversal of adverse cardiac remodeling.

More Information:

Please visit (NCT00893360) for more information on the completed CADUCEUS Phase I trial.