In short
Clinical trials of Antithrombin Iii Human are studying its use in people with hemophilia A or B and in people with antithrombin deficiency. These trials mainly look at safety, efficacy, and outcomes during surgery, delivery, or cardiac surgery. The studies include adults and patients with inherited or acquired antithrombin problems.
Key points
- Clinical trials of Antithrombin Iii Human are being done in people with hemophilia A or B, congenital antithrombin deficiency, and acquired antithrombin deficiency with heparin resistance. All listed studies are Phase 3 trials, so they are testing how well treatment works while also watching safety. One completed study looked at long-term safety and tolerability in hemophilia patients, including those with inhibitor antibodies. Another study is focused on patients with congenital antithrombin deficiency during surgery or delivery. A third study is looking at adults having cardiac surgery with cardiopulmonary bypass to see whether treatment restores and maintains heparin responsiveness.
Trial overview
Three clinical trials in the source data investigate Antithrombin Iii Human in different patient groups and clinical settings. All three are Phase 3 studies, which means they are larger trials that focus on how well a treatment works and continue to check safety. The studies include one completed trial and two authorised trials.
Hemophilia study
The completed trial with NCT ID 2023-508884-59-00 studied people with hemophilia A or hemophilia B, with or without inhibitory antibodies to factor VIII or IX. Inhibitory antibodies are proteins that can make treatment less effective because they block the clotting factors used in care. This study was an interventional Phase 3 trial with 281 participants.
The brief summary says the trial aimed to characterize the long-term safety and tolerability of fitusiran. The primary outcome was the number of participants with treatment-emergent adverse events, which means health problems that started or became worse after treatment began. The intervention list also included Antithrombin III, along with several clotting factor treatments used in the study context.
Congenital antithrombin deficiency study
The authorised trial NCT04918173 studied patients with congenital antithrombin deficiency who were undergoing surgery or delivery. Congenital means the condition is present from birth. This was a Phase 3 interventional study with 46 participants.
The study objective was to assess the incidence of a combined outcome made up of thrombotic events and thromboembolic events during treatment with Atenativ for surgical procedures or childbirth, and up to 30 days after treatment started. This endpoint looks at whether blood clots form or travel in the body during a high-risk period.
Cardiac surgery and heparin resistance study
The authorised trial NCT06096116 studied adult patients with acquired antithrombin deficiency who had heparin resistance and were scheduled for cardiac surgery needing cardiopulmonary bypass. Acquired means the condition develops later in life rather than being present from birth. This was also a Phase 3 interventional study with 142 participants.
The primary efficacy endpoint was the percentage of patients who did not need any further antithrombin-containing therapy, such as fresh frozen plasma or other antithrombin concentrates, to restore heparin responsiveness before bypass and to keep it during bypass. In simple terms, the study asked whether treatment could help heparin work well enough during heart surgery without extra rescue treatment.
Main outcomes and what they mean
The trials measure different outcomes depending on the patient group and the procedure being studied. In the hemophilia study, the main outcome was safety, measured by treatment-emergent adverse events. In the congenital deficiency study, the main outcome was clot-related events during surgery or delivery and for 30 days after treatment started. In the cardiac surgery study, the main outcome was whether heparin responsiveness could be restored and maintained without extra antithrombin therapy.
Who the trials are for
- People with hemophilia A or B: one completed trial studied patients with these bleeding disorders, including those with inhibitory antibodies to factor VIII or IX.
- People with congenital antithrombin deficiency: one authorised trial focused on patients with this inherited condition during surgery or delivery.
- Adults with acquired antithrombin deficiency and heparin resistance: one authorised trial studied adults scheduled for cardiac surgery with cardiopulmonary bypass.