PDA001 for the Treatment of Adults With Moderate-to-Severe Crohn's Disease

This was a Phase 2a, multicenter study designed to evaluate the safety, tolerability, and efficacy of intravenous human placenta-derived cells (PDA001) in adults with moderate-to-severe Crohn's disease. The study consisted of sequential cohorts. Initial open-label dose-escalation cohorts evaluated PDA001 administered intravenously at a dose of 1.6 × 10\^9 cells (8 units). A subsequent randomized, double-blind, placebo-controlled cohort evaluated PDA001 administered intravenously at doses of 200 × 10\^6 cells (1 unit) and 800 × 10\^6 cells (4 units) compared with placebo. The primary efficacy objective was to evaluate induction of clinical response, defined as a reduction from baseline of 25% and/or at least 100 points in the Crohn's Disease Activity Index (CDAI) at both Week 4 and Week 6. Secondary objectives included evaluation of clinical remission, safety, and tolerability. Subjects were followed during an induction phase and could participate in an extension phase with re-treatment according to protocol-defined criteria.