Active clinical trials for "Hemophilia A"

This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan. Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.

Emicizumab (Hemlibra®) is a subcutaneous hemostatic treatment approved in 2019 for the prophylaxis in severe hemophilia A. Emicizumab is a bispecific monoclonal antibody mimicking factor VIIIa that provides a constant level of coagulation similar to that observed in minor haemophilia A patients whose factor VIII level is 10-15%. Although the correction of plasma coagulation in vivo is only partial, emicizumab strongly shortens the in vitro coagulation times involving the intrinsic pathway such as aPTT(activated partial thromboplastin time). The investigators will evaluate the effect of emicizumab on a coagulation test (Activating clotting time (ACT i-STAT Alinity)) used as a point of care device to monitor heparin therapies during cardiac surgeries (cardiopulmonary bypass) and cardiac catheterizations. Because ACT is activated through the intrinsic pathway, it may also be shortened by emicizumab. Prophylactic treatment with emicizumab would make it impossible to use ACT for heparin therapy in a hemophiliac patient benefiting from this treatment. The aim of this in vitro study is to assess the effect of emicizumab on the in vitro heparin-induced ACT increase in severe hemophilia A patients treated with emicizumab and in healthy volunteers (measurement on the i- STAT Alinity) thanks to in vitro blood spiking experiments. Some data have already been published with other ACT devices (Hemochron..) but never with the i-STAT Alinity device which uses a different technology and other reagents.

Study MO42623 is a Phase IV, multicenter, open-label, three cohort study designed to evaluate the impact of emicizumab prophylaxis on overall health, physical activity, and joint outcomes in participants aged ≥13 and <70 years with severe hemophilia A without factor VIII (FVIII) inhibitors or moderate hemophilia A without FVIII inhibitors who are receiving FVIII prophylaxis and who will start emicizumab treatment as part of this study.

The aim of the HEM-POWR study is to understand better how Damoctocog alfa pegol (Jivi) is used to treat people with Hemophilia A in day-to-day life, how well the treatment is tolerated and how satisfied patients and physicians are with the treatment.

Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis

This is an open-label, multicenter Phase 1Ib/2II clinical trial of SS109 in adult hemophilia patients (≥ 18 years) with FVIII or FIX inhibitors to evaluate the PK/PD profile of SS109 and NovoSeven® after a single dose in adult hemophilia patients with FVIII or FIX inhibitors, to assess the preliminary efficacy and PK profile of SS109 during on-demand treatment, and to observe the safety and immunogenicity of SS109 throughout the study. The trial consists of three periods: screening period, PK study period, and on-demand treatment period. In the PK study period, subjects are divided into 2 cohorts (90 μg/kg and 270 μg/kg), which are sequentially conducted. Cohort 1 (90 μg/kg) enrollment is performed firstly, and Cohort 2 (270 μg/kg) enrollment is performed after Cohort 1 enrollment is completed. Subjects enter the PK study period as non-randomized. All screened eligible subjects will receive a single dose of comparator NovoSeven® in the absence of significant active hemorrhage, followed by PK/PD sample collection; then receive a single dose of the same dose of investigational drug SS109, followed by PK/PD sample collection. Specific times for PK/PD sample collection are listed in the schedule for biological sample collection. After completion of the PK study period, subjects will enter a 90-day on-demand treatment period and will be randomized into 3 groups (Group 1: 90 µg/kg, Group 2: 180 µg/kg, and Group 3: 270 µg/kg) at a ratio of 1:1:1. During on-demand treatment, subjects are treated on-demand with SS109 at the time of a new hemorrhage event and their efficacy is observed. The investigator will judge the severity of subject's hemorrhage according to the type, location, clinical symptoms and signs of the subject's hemorrhage. Appropriate hemostatic treatment regimens and whether or not to perform the first SS109 on-demand treatment for the hemorrhage event at home may be developed by the investigator based on the subject's on-demand treatment group, according to the severity of hemorrhage and the recommended dosing frequency of SS109 (see Dosage/Regimen), and the dosing interval may be adjusted in conjunction with the subject's response to treatment. If the subject's last hemostatic treatment is administered within one week before the D96 visit point during the on-demand treatment period, the subject is required to continue follow-up observation for one week after the last dose before completing the end of study visit. PK/PD samples will be collected as appropriate during on-demand treatment, as specified in the schedule for biological sample collection.Observe subject safety throughout the study.

International, post-authorisation non-interventional study to evaluate real-life effectiveness, safety and utilisation patterns of Octapharma's FVIII concentrates Nuwiq, Octanate, and Wilate in previously untreated and minimally treated severe haemophilia A patients in routine clinical practice.

The main aim of this study is to check for side effects and medical problems in participants with hemophilia A who are receiving ADYNOVATE in routine clinical practice. Also, it will be checked how effective ADYNOVATE is at stopping a bleed, at preventing bleeding episodes when used routinely for prevention and at controlling hemophilia-related bleeding during surgeries. Participants with hemophilia A who will be newly prescribed with ADYNOVATE treatment or have already been taking ADYNOVATE treatment according to the physician's decision will be included in this study. Participants will visit the study clinic as a part of their routine clinical practice and will be contacted by phone calls/home visits by home care workers for collection of treatment related data during the 6-month observation period.

A non-randomized, open-label, dose-escalation study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS802 in hemophilia A subjects with endogenous FVIII ≤2%.

A non-randomized, open-label, dose-escalation, phase I/II study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.