Abstract WP120: Welcoming the New Kid on the Block; The Phase IIb Prospective, Randomized, Open-Label, Blinded Endpoint (PROBE) Study of NanO 2 TM Neuroprotection in Large Vessel Strokes

Abstract

Background: NVX-208 (NanO 2 TM ) was recently examined in a completed randomized, placebo-controlled and double-blinded dose escalation Phase Ib/II safety trial in acute ischemic stroke (AIS) patients and was found to be safe in all dosage levels. Exploratory aims indicated early treatment in the highest NanO 2 TM dose cohort improved clinical outcomes of early NIHSS and 90-day mRS. In the current Phase 2b protocol, NanO 2 TM ’s enhanced oxygen delivery from the blood to tissue will be examined in early AIS subjects with large vessel occlusions (LVO). LVO patients will provide the optimal assessment for NanO 2 TM to maintain penumbra tissue viability. The Phase 2b primary objective will be to assess functional recovery and subject independence. Drug information: NanO 2 TM is an emulsion of 2% dodecafluoropentane (DDFP) in stabilizers (sucrose, PEG-Telomer-B) and phosphate buffered saline (pH 7.0) that is a highly efficient fluorocarbon oxygen transporter. Compared to previously developed fluorocarbon oxygen carriers, NanO 2 TM carries far more oxygen per gram of fluorocarbon. Because DDFP is not metabolized, almost 100% of administered doses was recovered in the subject’s breath as DDFP. Hypothesis: NanO 2 TM given early to subjects with LVO ischemic stroke will maintain penumbra tissue viability. Methods: Phase 2b study sites will include multiple stand-alone and hub and spoke systems located across the United States. Central IRB and safety monitors will provide oversight and support coverage. Key methodology includes providing early first dosage administration of NanO 2 TM or placebo (study treatment) to identify and consent LVO AIS subjects with viable penumbra. For drip and ship patients, second and third doses will be given en route or after arrival at the hub hospital and after revascularization procedures, respectively. Following the third dose, all subjects will receive study treatment dosing out to 24 hours, each dose at 90 minute intervals. 24-hour imagery assessments will confirm infarct volume. All final angiograms and 24-hour imaging data will be sent to core labs for blinded confirmatory review. Subject recovery and independence will be assessed throughout the study to 90 days. Conclusion: Study projected start date is early to mid-2020.