Final Operational Milestone #5
A Phase 1 a/b clinical study was conducted in which cardiosphere-derived cells (CDCs) were infused into patients with pulmonary arterial hypertension (PAH). Phase 1a (N=6) was open-label for dose escalation purposes. Phase 1b was a double-blind placebo-controlled study in which half (N=10) were infused with maximum dose CDCs (100 million cells) while the other half (N=10) received placebo. Extensive studies were performed over a period of a year and several complex tests were repeated at four months. Short and long term safety was carefully monitored throughout the duration of the study. No significant safety issues were observed. Several encouraging exploratory efficacy endpoints were noted across several different testing variables.
A Clinical Study Report (CSR) was generated based on the clinical objectives of the protocol.