Date: 2012-07-10
Type of information:
phase: 3
Announcement: hold of two clinical trials
Company: Inspiration Biopharmaceuticals (USA) Ipsen (France)
Product: IB1001 (recombinant factor IX)
Action mechanism:
Disease: hemophilia B
Therapeutic area: Hematological diseases - Genetic diseases
Country: USA
Trial details:
Latest
news: * On July 10, 2012, Ipsen has announced that its partner Inspiration Biopharmaceuticals was notified by the FDA that both clinical trials evaluating the safety and efficacy of IB1001 were placed on clinical hold. The clinical hold impacts two ongoing IB1001 clinical trials – a phase III study evaluating the safety and efficacy of IB1001 to treat and prevent bleeding episodes in adults with hemophilia B, and a phase III study evaluating the safety and efficacy of IB1001 to treat and prevent bleeding episodes in previously treated pediatric subjects with hemophilia B. The adult study has completed its primary analysis period.
Following the FDA's request, Inspiration has notified clinical sites in the U.S. to hold treatment of patients with IB1001. Inspiration is also sharing the FDA directive with regulators in countries outside of the U.S. where the studies are being conducted.
During the course of routine laboratory evaluations conducted as part of the ongoing phase III clinical trials, Inspiration observed, and reported to the FDA, a trend towards a higher proportion of IB1001 treated individuals developing a positive response to testing of antibodies to Chinese Hamster Ovary (CHO) protein, the product's host cell protein (HCP). Small amounts of host cell protein are expected and documented in recombinant therapeutic products of all types. Nevertheless, the higher than expected rate of anti-CHO antibody development in people treated with IB1001 has led Inspiration to initiate a thorough investigation.
A total of 86 people with hemophilia B have received IB1001 in clinical studies and, to date, no adverse events (anaphylaxis or other serious allergic type reaction and nephrotic syndrome) related to the development of antibodies to CHO protein have been reported. Furthermore, no relationship has been demonstrated between the development of antibodies to CHO protein and the development of any antibodies to factor IX. Inspiration continues to follow subjects enrolled in clinical trials of IB1001 to collect safety-related information and will share this information with regulators. While this finding may be a potential safety concern, no evidence suggests a change in the current overall clinical benefit and risk profile of IB1001.
* On February 22, 2012, Inspiration Biopharmaceuticals has announced clinical results its intravenous recombinant factor IX (FIX) product being investigated for the treatment and prevention of bleeding in individuals with hemophilia B. The data demonstrated that Inspiration\'s FIX product provided effective surgical hemostasis in people with hemophilia B undergoing major surgical procedures. The data were highlighted in a poster presentation at the 5th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) in Rome, Italy. In a poster titled, \"Use of IB1001, a new investigational recombinant factor IX, in patients with hemophilia B undergoing major surgical procedures\", the newly reported data showed that a group of subjects who underwent major surgical procedures had effective control of bleeding with IB1001. Estimated intra-operative blood loss was rated as expected or less than expected in all subjects, and IB1001 was well-tolerated with few adverse events.
Fourteen subjects, ages 12 and above, were enrolled in the study. Twelve underwent orthopedic procedures, with one inguinal repair and one hysterectomy. Subjects received either bolus or continuous infusion of IB1001 to support surgery, and for a minimum of three days post procedure. Hemostasis was evaluated at 12 hours and 24 hours post procedure. Post-surgical hemostasis was considered adequate or superior at both evaluation points in all subjects.
* On February 4, 2011, Inspiration Biopharmaceuticals has presented pharmacokinetic data on its lead product, IB1001, a recombinant factor IX (FIX) for the treatment and prevention of bleeding in individuals with hemophilia B. According to Inspiration, results of the Phase 1 portion of an ongoing IB1001 clinical study demonstrated non-inferiority of IB1001 in achieving overall levels of replacement factor compared to BeneFIX®, the only approved recombinant FIX product for the treatment of hemophilia B. Currently, IB1001 is in Phase 3 and safety and efficacy results are expected later this year.
