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 September 1, 2014

Actavis released results from two phase III studies of ceftazidime-avibactam as a treatment for adult hospitalized patients with complicated intra-abdominal infections. RECLAIM-1 and -2 are phase III, randomized, multi-center, double-blind, double-dummy, parallel-group, comparative studies to determine the efficacy, safety and tolerability of ceftazidime/avibactam (2000mg/500mg, q8h) plus metronidazole (0.5g q8h) versus meropenem (1g q8h) in the treatment of complicated intra-abdominal infections in hospitalized adults. A total of 1,066 patients have been randomized to the RECLAIM-1 and -2 trials from 30 countries.

Amicus Therapeutics issued results of a phase III trial of migalastat HCl for the treatment of Fabry disease with amenable mutations. The study compared oral migalastat to standard-of-care enzyme replacement therapies (ERTs) for Fabry disease (Fabrazyme and Replagal). The study enrolled 60 patients (26 males and 34 females) with Fabry disease with amenable mutations in a clinical trial assay who had been treated with ERT for a minimum of 12 months prior to study entry. These patients were randomized 1.5:1 to switch to migalastat (36 patients) or remain on ERT (24 patients) for the primary 18-month treatment period, after which they were eligible to receive migalastat in a 12-month extension phase. Among the 60 patients enrolled, 56 (34 in the migalastat group and 22 in the ERT group) had amenable mutations in a GLP-validated human embryonic kidney (HEK) cell-based in vitro assay (GLP HEK-amenable). Migalastat had a comparable effect to ERT on patients’ kidney function as measured by the change in eGFR and mGFR. Levels of plasma lyso-Gb3, an important biomarker of disease, remained low and stable in patients with amenable mutations who switched from ERT to migalastat. Of 48 patients with GLP HEK-amenable mutations who completed the study, 46 (96%) elected to continue with the 12-month treatment extension and 45 remain on migalastat as their only treatment for Fabry disease. Migalastat was generally safe and well-tolerated.

Baxter International reported results of a phase III clinical trial of BAX 855 for treatment of treatment for hemophilia A. The global, multi-center, open-label study evaluated BAX 855 among 138 adolescent and adult patients with previously-treated hemophilia A. Patients received treatment twice weekly (45IU/kg) or on-demand, and were followed for six months. The study demonstrated that BAX 855 met its primary endpoint in the control and prevention of bleeding, routine prophylaxis and perioperative management for patients 12 years or older. Patients in a twice-weekly prophylaxis arm experienced a 95% reduction in median ABR as compared to those in the on-demand arm (1.9 v. 41.5, respectively). BAX 855 also was effective in treating bleeding episodes, 96% of which were controlled with one or two infusions. The half-life of BAX 855 was 1.4 to 1.5 times that of ADVATE, consistent with the findings from the phase I study. No patients developed inhibitors to BAX 855 and no treatment-related serious adverse events, including hypersensitivity, were reported. The most common (three patients) productrelated adverse event was headache. Baxter expects to submit a BLA for BAX 855 to the FDA before the end of 2014.

Biotronik reported results of a worldwide, randomized, controlled trial of Biotronik Home Monitoring for the detection of heart failure at an early stage. 274 patients received an ICD and 390 patients received a CRT-D in adherence with European guidelines, and were followed for one year. The primary outcome, worsening heart failure, was based on a composite score including death, hospitalization, NYHA class and patient self-assessment. Secondary outcomes included all-cause mortality and hospitalization. Home Monitoring reduced all-cause mortality by over 50%. Only 18.9% of patients using Home Monitoring experienced the worsening of heart failure, compared to 27.2% in the control arm (p=0.013). The prospective study randomized 664 patients with chronic heart failure, NYHA class II or III symptoms, ejection fraction ≤35%, and optimal drug therapy in groups with or without telemonitoring in a 1:1 ratio.

Eli Lilly issued results of a phase III study of ixekizumab for moderate-to-severe plaque psoriasis. In the three UNCOVER studies, 3,866 patients were assigned to receive either placebo or ixekizumab (80mg every two or four weeks) for 12 weeks, following a 160mg starting dose. In the two active comparator studies (UNCOVER-2 and 3), patients could be assigned to receive etanercept 50mg twice weekly for 12 weeks. In UNCOVER- 1, responders to treatment were assigned to continue treatment on either placebo or ixekizumab (80mg every four or 12 weeks) for up to 60 weeks. For patients treated with ixekizumab either every four weeks or every two weeks, between 78% to 90% of patients achieved at least a 75% reduction in Psoriasis Area and Severity Index (PASI) score (PASI 75) at 12 weeks. Additionally, 31% to 41% of these patients achieved PASI 100, or clear skin, at week 12. For comparison, between 5% and 7% of patients treated with etanercept in the UNCOVER-2 and 3 studies achieved PASI 100. The company intends to submit ixekizumab to regulatory authorities in the first half of 2015.