AMI Phase I Trial Design
AMR–001: First stem cell product to demonstrate in a clinical trial a dose–related improvement in cardiac perfusion
In a Phase I study of 31 patients, AMR–001 showed a dose–related significant improvement in perfusion. Presented
at the 2009 American College of Cardiology Annual Scientific Session, the Phase I study results demonstrated that patients receiving
10 and 15 million cells (n=9) showed significant improvement in resting perfusion rates at six months as compared to patients receiving
5 million cells (n=6) and control (n=15), as measured by the SPECT total severity score, (–256 versus +13, p=0.01). The data also showed
that patients receiving 10 or more million cells showed a trend towards improvement in ejection fraction, the percentage of blood pumped
out of a ventricle with each heartbeat, (+4.5% versus +0.69%); end systolic volume (–5.7mL versus +3.5mL); and infarct size, tissue death
due to loss of adequate blood supply, (–7.4% versus –5.3%) at six month follow–up. No study–related significant adverse
events were reported.
Summary of the Phase I Clinical Trial Protocol
Indication |
Post-AMI with LVEF ≤50% and Wall Motion Abnormality in the myocardium of the IRA |
Primary End Point |
Safety in post-AMI Patients |
Other Endpoints |
RTSS (Perfusion); LVEF; ESV; SDF Mobility |
Key Inclusion Criteria |
Confirmation of ST Elevation MI; Ejection fraction ≤50% |
Dosing Frequency |
Single Dose |
Groups and Randomization |
3 dose cohorts (5, 10, 15 Million cells, randomized 1:1) |
Number of Subjects |
N=31 |
Number of Sites |
4 |
Geography |
United States |
Trial Duration |
6 months |
Source: Quyyumi AA et al 2011, American Heart Journal; 161(1) 98-105
PreSERVE AMI Trial - Phase II Clinical Plan
The Phase II trial has been designed to evaluate the potential of AMR-001 to improve perfusion, preserve cardiac function and improve clinical outcomes. The trial is expected to include 160 patients in a placebo controlled, double-blind study. A composite of cardiac measures, including clinically meaningful endpoints, will support the primary endpoint of improved cardiac perfusion.
Indication |
Post-AMI Preservation of Cardiac Function |
Primary Endpoint |
Increased Cardiac Perfusion (RTSS) measured by SPECT |
Other Endpoints |
A composite of endpoints will be used to determine overall cardiac function (including preservation of LVEF and prevention of adverse remodeling) and Quality of Life (KCCQ & SAQ*) |
Safety |
Reduction in cumulative MACE and other adverse events at 6, 12, 18, 24, and 36 months |
Dosing Frequency |
Single dose |
Dosing and Randomization |
Minimum dose for release >10 M cells
Randomized 1:1 treatment to sham placebo control |
Number of Subjects |
160 patients |
Number of Sites |
34 |
Geography |
United States |
Trial Duration |
Perfusion, cardiac function and QOL at approximately 18 months post first enrollment (12 months of enrollment and 6 months of treatment) |
* KCCQ: Kansas City Cardiomyopathy Questionnaire
SAQ: Seattle Angina Questionnaire
