ApcinteX will conduct a global prospective cohort study with Centessa Pharmaceuticals to assess annualized bleeding rates and standard treatments among patients with severe hemophilia A or B.

The noninterventional study’s primary outcome measures are annualized bleeding rates for treated bleeds and all bleeds per participant, and average therapeutic doses for bleeding episodes by product, per time period, and per bleeding episode (minimum time period: 12 weeks).

Up to 200 male patients aged 12 to 65 with historically documented severe hemophilia A or B, with or without inhibitors and who are treated with standard on-demand or prophylactic therapy will be eligible to enroll. Patients with thrombophilia or severe obesity, previous deep vein thrombosis, or a history of intolerance to subcutaneous injections, among other exclusion criteria, are not eligible to enroll.


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Enrolled patients may be eligible to participate in future clinical interventional studies planned by the sponsor.

Read more about hemophilia treatment

Hemophilia is a rare genetic disorder that involves a lack of blood clotting factors, resulting in spontaneous bleeding episodes and bleeding after injures or surgeries that cannot always be controlled. Patients with hemophilia are mostly male, and current standard treatments involve frequent and time-consuming intravenous administrations of replacement clotting factors. In hemophilia A, the missing clotting factor is known as VIII and in hemophilia B it is known as factor IX.

Observational studies do not assess the safety and efficacy of any particular medicine or therapy. Instead, they provide information on health outcomes, “real world” use of treatments, and about risks and benefits of treatments as observed in patients. These studies can also lead to research questions to be examined in future interventional clinical trials and they can inform daily clinical practice with a specific patient group.

The study’s estimated start date is December 2022 and its approximate completion date is November 2024.

Reference

Severe hemophilia non-interventional study. ClinicalTrials.gov. November 4, 2022. Accessed November 13, 2022.