Clinical Trial Resource Center

Clinical Trial Resource Center

New Medical Therapies™

Latest New Medical Therapy Trial Results

Following are the most recent results for new drug therapies currently in clinical trials worldwide. Results are also searchable by therapeutic area beginning with the most recent updates.

Week of March 23, 2015

CSL Behring issued results of a phase III study of Kcentra for the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (VKA) therapy in adult patients needing an urgent surgery or invasive procedure. Results of the multicenter, open-label, phase IIIb randomized trial showed in 168 evaluable patients, 90% of patients treated with Kcentra achieved effective hemostasis, compared to 75% of patients treated with plasma. Additionally, INR reduction to <=1.3 at 30 minutes after the end of infusion was achieved in 55% of patients treated with Kcentra v. 10% of patients treated with plasma. Incidences of adverse events, serious adverse events, thromboembolic events and deaths were similar between the Kcentra and plasma groups. In post-hoc analysis, the median time from start of infusion to start of urgent surgical procedure was shorter in the Kcentra group (3.6 hours [IQR 1.9-10.8]) than in the plasma group (8.5 hours [IQR 2.8-18.7]); (p=0.0098).

CTI BioPharma and Baxter International released results of a phase III study of pacritinib for primary or secondary myelofibrosis. The randomized (2:1), open-label, multinational trial enrolled 327 patients and demonstrated that pacritinib treatment provided a clinically and statistically significant response rate (p=0.0003) in spleen volume reduction in patients with myelofibrosis when compared to physician-specified best available therapy (BAT). Importantly, the trial results also demonstrated a significant difference among patients with platelet counts of less than 100,000 per microliter and less than 50,000 per microliter, both subgroups that were stratified at randomization. The magnitude of treatment effect was consistent with previously reported phase II results, with the greatest reduction observed among the sickest patients (platelet counts <50,000 per microliter). Among 50 patients who were red blood cell (RBC) transfusion dependent at study entry (six units of RBC over 90 days pre-entry), pacritinib therapy resulted in a clinically meaningful percentage of patients becoming transfusion independent compared to BAT. 79% of patients in the BAT arm of the study crossed over to pacritinib therapy. The safety profile was consistent with prior phase II trials. The PERSIST clinical trials are intended to support an NDA to the FDA.

Ocular Therapeutix reported results of a phase III study of OTX-DP (Sustained Release Dexamethasone) for ocular inflammation and pain following cataract surgery. The multicenter, randomized, parallel-arm, double-masked, vehicle-controlled study enrolled 247 patients randomized 2:1 to receive either OTX-DP or a placebo vehicle control punctum plug without active drug. 33.7% of OTX-DP-treated patients showed an absence of inflammatory cells in the anterior chamber of the study eye on day 14 following drug product insertion, compared to 14.6% of those receiving placebo vehicle control punctum plug (p=0.0015). In addition, 76.1% of patients receiving OTX-DP reported absence of pain in the study eye on day 8 following insertion of the drug product, compared to 36.1% of those receiving placebo vehicle control punctum plug (p<0.0001). Ocular is continuing to analyze the safety findings from the clinical trial. The company is on track to submit an NDA to the FDA for OTX-DP for post-surgical ocular inflammation and pain in the second quarter of 2015.

Regeneron Pharmaceuticals and Sanofi issued results of a phase III trial of Praluent (alirocumab) involving 2,341 high-risk patients with hypercholesterolemia. The 78-week trial evaluated Praluent 150mg (n=1,553) every two weeks compared to placebo (n=788). At week 24, Praluent reduced LDL-C from baseline by an additional 62% v. placebo (p<0.0001) when added to the current standard-of-care, which included maximally-tolerated statins. Efficacy remained consistent throughout treatment, and at week 78 there was a 56% reduction from baseline in LDL-C for Praluent v. placebo (p<0.0001). At week 24, 81% of patients in the Praluent group achieved their pre-specified LDL-C goal (either 70mg/ deciliter [mg/dL] or 100mg/dL depending on baseline CV risk) compared to 8.5% for placebo (p<0.0001). Adverse events (AEs) occurred in 81% of Praluent and 83% of placebo patients, leading to discontinuation in 7.2% and5.8% of patients, respectively. Earlier this year, Regeneron and Sanofi announced the FDA accepted the BLA for Praluent for priority review.