A Study of Elranatamab for Inhibitor Eradication in Patients With Hemophilia A
Study Details
- Hemophilia A With Inhibitor(s)
- Drug: Elranatamab
- Male or female patients with moderate-to-severe hemophilia A (factor VIII activity \<2%).
- Aged 18 to 65 years, inclusive.
- Positive factor VIII inhibitor detected on at least two consecutive occasions (inhibitor titer \>0.6 BU/mL).
- Baseline factor VIII inhibitor titer \>10 BU/mL at the time of enrollment.
- Known hypersensitivity to Elranatamab or any of its excipients.
- Presence of other autoimmune diseases or a need for immunosuppressive therapy for reasons unrelated to this study.
- History of malignancy within 5 years prior to screening, with the exception of adequately treated carcinoma in situ of the cervix or non-metastatic cutaneous squamous cell carcinoma or basal cell carcinoma.
- History of severe recurrent or chronic infections, or acute infection requiring systemic treatment with antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungal agents within 4 weeks before the first dose or during the screening period, or superficial skin infection requiring systemic therapy within 1 week before the first dose.
- Clinically significant laboratory abnormalities at screening, including:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3 × the upper limit of normal (ULN).
- Total bilirubin \>1.5 × ULN (subjects with documented Gilbert syndrome are not excluded based on this criterion);
- Absolute neutrophil count \<1,500/mm³;
- Hemoglobin \<9 g/dL or immunoglobulin G (IgG) \<500 mg/dL;
- Absolute lymphocyte count \<500/mm³;
- Creatinine clearance (CrCl) \<30 mL/min.
- Positive test for HIV antibody or syphilis antibody.
- Positive hepatitis B surface antigen (HBsAg); or positive hepatitis B core antibody (anti-HBc) with detectable HBV DNA by polymerase chain reaction (PCR). Subjects positive for hepatitis C virus (HCV) antibody are excluded.
- Women who are pregnant or breastfeeding, or who plan to become pregnant or breastfeed during the study; men whose partners plan to become pregnant during the study.
- Subjects with psychiatric disorders that impair their ability to provide informed consent or comply with study procedures and follow-up.
- Subjects with unresolved toxicities from prior therapies before study participation.
- Any other condition that, in the opinion of the investigator, would make the subject unsuitable for participation in the study.
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.