Clinical Trials Resource Center


April 4, 2016

Pfizer issued results of a phase III study of a once-weekly regimen of BeneFIX Coagulation Factor IX (recombinant) 100IU/kg prophylaxis versus on-demand treatment in people with moderately severe or severe hemophilia B. This was an international, non-randomized, open-label sequential-period trial with a six-month period of on-demand treatment followed by 12-month period of prophylaxis, with a mean total therapy duration of 550 days. All 25 participants had moderately severe or severe hemophilia B (factor IX activity 2 IU/dL or less) and were male with a mean age of 31.3 years; participants had experienced at least 12 bleeding events, six of them in joints, in the previous year. All participants received BeneFIX, and no one discontinued treatment early. The study showed that the median ABR (annualized bleeding rate) values were 2 (range, 0-13.8) for the prophylaxis period and 33.6 (range, 6.1-69.0) for the on-demand period. The mean ABR values were 3.6 ± 4.6 for the prophylaxis period and 32.9 ±17.4 for the on-demand period, which were statistically significantly different (p<0.0001). During the 52 weeks of the prophylaxis period, 36% of patients experienced no bleeding events of any kind, and 48% of patients experienced no spontaneous bleeding events. Almost half (47%) of the 17 factor IX activity levels measured approximately one week postdose were greater than 2 IU/dL (2.1-10.4IU/dL), and three patients had zero ABR despite a factor IX activity level of zero. In the study, the adverse events reported for the prophylactic regimen were similar to those reported for on-demand treatment. The most common adverse events, occurring in 10% or more of patients during either regimen, were arthralgia, back pain, headache, joint swelling, local swelling, nasopharyngitis, pharyngitis, pyrexia, toothache and upper respiratory tract infection. Neither inhibitors nor thrombotic events were reported.

August 24, 2015

Biogen has issued results of a phase III study of Alprolix for severe hemophilia B. B-YOND enrolled 116 males, including 93 participants (81%) who completed B-LONG, and 23 (100%) of those who completed Kids B-LONG. The open-label extension study was 27.6 months for adults and adolescents and 47.7 weeks for children under age 12. The median overall median annualized bleeding rates (ABR) was zero for children under age 6 who received weekly prophylaxis (n=9). For children between 6 and 12 years old, median overall ABRs were 2.65 (n=10), 2.37 (n=5) and 3.13 (n=1) in weekly, individualized and modified prophylaxis regimens, respectively. In each age group, the median average weekly dose for participants previously on prophylaxis was similar for individuals in the weekly and individualized treatment arms. Safety results were typical of the hemophilia B populations studied. The most common adverse events (incidence of greater than or equal to 5 percent) for adults and adolescents included headache, common cold and vomiting. For children under age 12, the most common adverse events were falls, common cold and seasonal allergy.

March 9, 2015

Biogen Idec and Swedish Orphan Biovitrum released results of a phase III, global, open-label, multicenter trial of Alprolix in 30 boys under age 12 with severe hemophilia B. In the study, children treated prophylactically with Alprolix had an overall median annualized bleeding rates (ABR) of 1.97. The median ABR for spontaneous joint bleeds was zero. Approximately 33% of participants in the study experienced zero bleeding episodes. Overall, 91.7% of bleeding episodes were controlled by one or two infusions of Alprolix. The terminal half-life of Alprolix in the study was 66.5 hours for children under six and 70.3 hours for children six to less than 12 years of age. Alprolix was generally well-tolerated and no cases of serious allergic reactions or vascular thrombotic events were reported. These data will enable regulatory filings in Europe later this year as well as support pediatric indications in other countries.

February 23, 2015

Baxter International reported results of a phase III study of BAX 855 for hemophilia A. The prospective, global, multicenter, openlabel, two-arm phase III study evaluated BAX 855 among 137 previously treated hemophilia A patients (PTP) who were 12 years or older. Patients were assigned either to twice weekly prophylaxis (40-50IU/kg, n=120) or on-demand treatment (10-50IU/kg, n=17). In addition to a reduced annualized bleed rate (ABR), BAX 855 also was effective in treating bleeding episodes, 96% of which were controlled with one or two infusions at a median dose of 29IU/ kg per infusion. Treatment was rated excellent or good for nearly all episodes (96.2%). In the prophylactic group (n=101), 40% of patients experienced no bleeds. No patients developed inhibitors to BAX 855 and no treatment-related serious adverse events, including hypersensitivity, were reported. Seven adverse reactions in six patients, including headache, diarrhea, nausea and flushing were reported. The study findings supported Baxter’s December 2014 submission for approval of BAX 855 to the FDA. Baxter’s continuation study for patients who completed the pivotal trial and the phase III study among previously treated patients under the age of 12 with severe hemophilia A remain ongoing. Upon completion of the pediatric study, Baxter expects to file for marketing authorization with the EMA in 2016.

September 1, 2014

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.

April 21, 2014

Biogen Idec and Swedish Orphan Biovitrumreported results of a phase III trial of ELOCTATE for the treatment of children under 12 with severe hemophilia A. The global, open-label, multi-center study involved 71 boys with severe hemophilia A (factor VIII activity less than 1IU/dL, or 1%) with at least 50 prior exposure days to factor VIII therapies. The study was conducted at 23 hemophilia treatment centers in eight countries. Overall, 67 participants (94%) completed the study (33 under six years old and 34 six-to-11 years old). The average time participants spent in the study was 25 weeks, and 61 participants received ELOCTATE infusions on at least 50 separate days (exposure days) to assess inhibitor development. All study participants were to be initially treated with twice-weekly prophylactic infusions of ELOCTATE [25IU/kg day one, 50IU/kg day four]. Study investigators could adjust the dose or interval based on individual response. Approximately 90% of study participants were on twice-weekly dosing at the end of the study. The relative increase in half-life in children with severe hemophilia A was consistent with the 1.5-fold increase in half-life seen in the A-LONG study of adults and adolescents. Children treated prophylactically with ELOCTATE had an overall median ABR of two and a median ABR for spontaneous bleeds of zero. Forty-six percent of participants in the study experienced zero bleeding episodes. Overall, 93% of bleeding episodes were controlled by one to two infusions of ELOCTATE. ELOCTATE was generally well-tolerated. This is considered a milestone that will enable regulatory submission in Europe.

August 5, 2013

rEVO Biologics issued results of a multi-center study of LR769, a novel recombinant form of human Factor VIIa (rhFVIIa), for the treatment of hemophilia A and B patients with inhibitors to Factor VIII or IX. Conducted in the U.S. and Europe, LR769 was administered in three dose levels— 25μg/kg, 75μg/kg and 225μg/kg, with each patient receiving two doses. LR769 is pharmacodynamically active after a single dose, and dose-related effects on core coagulation markers (aPTT, PT, and F1+2) were found. LR769 is well-tolerated in hemophilia A or B patients when administered at doses up to 225μg/kg and no immune reactions to LR769 were found in any patients after repeated dosing. There are plans for registrational trials later this year.

October 1, 2012

Biogen Idec and Swedish Orphan Biovitrum released results from a phase III trial of rFIXFc for the treatment of hemophilia B. This open-label, multi-center, multi-arm study, B-LONG, enrolled 123 male patients aged 12 years and older. Subjects were divided into four arms and received rFIXFc 50IU/kg weekly as prophylaxis, rFIXFc 100IU/kg every 10 days as an individualized interval prophylaxis, episodic treatment or perioperative management. Data showed that the overall median annualized bleeding rates (including spontaneous and traumatic bleeds) were 2.95 in the weekly prophylaxis arm; 1.38 in the individualized interval prophylaxis arm; and 17.69 in the episodic treatment arm. In the individualized interval prophylaxis arm, the median dosing interval during the last six months on study was 14 days. Overall, 90.4% of bleeding episodes were controlled by a single injection of rFIXFc. The drug was well tolerated. The most frequent adverse events were nasopharyngitis, influenza, arthralgia, upper respiratory infection, hypertension and headache. Based on these findings, Biogen Idec plans to submit a Biologics License Application to the FDA in the first half of 2013. Additional analyses of the B-LONG study are ongoing.

July 16, 2012

Inspiration Biopharmaceuticals reported interim results from a phase II/III trial of OBI-1 for the treatment of acquired hemophilia A. The open-label study enrolled seven patients experiencing serious bleeds. Subjects received 200U/kg of OBI-1 on first injection and were subsequently dosed based on target FVIII levels. All seven subjects experienced successful control of bleeds at 24 hours and subsequent resolution of their bleeds. Therapeutic FVIII activity levels were achieved and maintained with intermittent OBI-1 administration based on FVIII levels. Of the adverse events reported during the study thus far, five were serious, but these were not considered by investigators to be treatment-related. Inspiration Biopharmaceuticals plans to continue the phase II/III trial.

February 27, 2012

Inspiration Biopharmaceuticals reported results from a clinical trial evaluating IB1001 for hemophilia B. The trial enrolled 14 subjects ages 12 and older with hemophilia B who were scheduled for major surgical procedures. The subjects received either bolus or continuous infusion of IB1001 during surgery and for a minimum of three days post procedure. Hemostasis was evaluated at 12 hours and 24 hours post procedure. IB1001 resulted in effective control of bleeding. Estimated intra-operative blood loss was rated as expected or less than expected in all subjects. IB1001 was well-tolerated with few adverse events.

July 19, 2010

Biogen Idec and Swedish Orphan Biovitrum issued positive results from a phase I/II trial of their fully-recombinant factor IX Fc fusion protein, rFIXFc, for the treatment of hemophilia B. This open-label, multi-center, dose-escalation study, SYN-FIXFc-07-001, enrolled 14 previously-treated subjects with severe hemophilia B. The subjects received a single dose of rFIXFc given as an intravenous injection at doses ranging from 1 to 100 IU/kg. rFIXFc was well tolerated and demonstrated an approximately three-fold increase in half-life compared to historical data for existing therapies.

August 29, 2005

Baxter Healthcare reported positive results from a multi trials investigating Advate, a recombinant antihemophilic factor for the treatment of hemophilia A. The first results were from a preliminary assessment of a prospective international, multi-center study enrolling 53 pediatric subjects less than six years of age who were previously treated with factor VIII. Results demonstrated that no patients discontinued therapy as a result of an adverse event and no incidence of factor VIII antibodies were reported in this study. The second were of preliminary results of a multi- center, prospective, open-label, uncontrolled study designed to test the efficacy, safety and immunogenicity on 57 hemophilia A subjects undergoing surgery or other invasive procedures. An interim assessment of the first 44 procedures performed demonstrated an efficacy rating of excellent/good in 97.7% of surgeries. The company presented the data at the 20th Congress of the International Society on Thrombosis and Haemostasis held in Sydney, Australia.

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