Emicizumab in Patients With Acquired Hemophilia A
Study Details
- Hemophilia A, Acquired
- Drug: emicizumab
- Inclusion Criteria:
- Signed Informed Consent/Assent Form
- Age ≥18 years at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Diagnosis of AHA based on a reduced FVIII activity (\<50 %) and positive FVIII inhibitor (\>0.6 BU/ml) at screening (local laboratory)
- Current bleeding due to AHA at the time of screening
- Plan to be adherent to emicizumab prophylaxis during the study
- For women of childbearing potential who meet the following criteria:
- Refrain from heterosexual intercourse or use contraceptive methods that result in a failure rate of \<1% per year during the study period A woman with ≥ 12 continuous months of amenorrhea with no identified cause other than menopause and has not undergone surgical sterilization (removal of ovaries and/or uterus). use of combined oral or injected hormonal contraceptive, bilateral tubal ligation, male sterilization, hormone- releasing intrauterine devices, and copper intrauterine devices.
- Exclusion Criteria:
- Congenital hemophilia A
- Treatment with aPCC within the last 24 hours before first study treatment or planned treatment with aPCC during the course of the study
- Known positive lupus anticoagulant at the time of screening
- Severe uncontrolled infection at the time of screening
- Signs of active disseminated intravascular coagulation at the time of screening -
- Emicizumab ⎯ AHA Emi Version 1.0 20
- Current treatment for thromboembolic disease or signs of current thromboembolic disease at time of screening
- Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator's judgment
- Known severe congenital or acquired thrombophilia
- Life expectancy \<3 months at the time of screening
- Other conditions that substantially increase risk of bleeding or thrombosis by the discretion of the investigator
- Contraindications according to the Investigator's Brochure of emicizumab
- Current treatment with emicizumab at time of screening
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection by the discretion of the investigator
- Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the local investigator, preclude the patient's safe participation in and completion of the study
- Addiction or other diseases that preclude the patient from appropriately assessing the nature and scope as well as possible consequences of the clinical study by the discretion of the investigator
- Pregnant or breast-feeding women
- Would refuse treatment with blood or blood products, if necessary.
- Subject is in custody by order of an authority or a court of law
- Treatment with any of the following:
- An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration before Study Day 1
- A non-hemophilia-related investigational drug within the last 30 days or 5 half-lives- before Study Day 1, whichever is longer
- An investigational drug concurrently
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
Protocol Summary
This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.