Abstract 71: Final Results of the B01-02 Phase 2 Trial Testing the Safety and Efficacy of MultiStem® in Treatment of Ischemic Stroke

    loading  Checking for direct PDF access through Ovid


Introduction: MultiStem is an adult, adherent, stem cell product, having shown safety and efficacy in other clinical indications and pre-clinical models of stroke. We assessed whether it was safe and improved outcomes in patients with ischemic stroke.

Methods: B01-02 was a double-blind, placebo-controlled study of ischemic stroke patients (NIHSS 8-20, inclusive) treated within 24-48 hours of symptoms at 33 sites in the U.S. and U.K. Patients were randomized 1:1 and received infusion of 1.2 billion cells or placebo. Efficacy endpoints included Global Recovery (mRS ≤2, NIHSS Δ ≥75% and BI ≥95) and Excellent Outcome between groups (mRS ≤1, NIHSS ≤1, BI ≥95) at Day 90, among others. Safety end points included neurologic worsening, secondary infections, adverse events and mortality.

Results: 126 patients formed the Intention-To-Treat (ITT) population, 65 receiving MultiStem, 61 placebo. The cell therapy did not show a significant benefit relative to placebo for the primary and secondary endpoints. However, MultiStem treatment was associated with lower rates of infections and pulmonary events, a reduction in hospitalization, and a reduction in life threatening adverse events and death. A higher proportion of patients receiving MultiStem treatment achieved an Excellent Outcome (p=0.10) compared to placebo. Post-hoc analyses indicate that, compared to placebo subjects (n=52), patients who received cell treatment earlier in the treatment window (≤36 hrs, n=27) had better Global Recovery (41.9% vs. 17.3%, p<0.01) and Excellent Outcome (18.5% v. 3.8%, p=0.03), had significantly better recovery by mRS shift analysis (p=0.03) and significantly reduced hospitalization (6.7d vs. 10.3 d, p<0.01).

Conclusions: Administration of MultiStem is safe and well tolerated in ischemic stroke patients within 48 hours of onset and reduces adverse events and death. Post-hoc analyses suggest that earlier administration of MultiStem may provide therapeutic benefit. Additional clinical studies exploring the optimal time frame for MultiStem administration are warranted.

Related Topics

    loading  Loading Related Articles