Hemlibra in Mild Hemophilia A
Study Details
- Factor VII Deficiency
- Drug: Emicizumab
- Inclusion Criteria:
- Signed informed consent form from the subject, parent or guardian
- Male sex
- Diagnosis of mild congenital hemophilia A (baseline FVIII level of \>5% to 30%) without a current FVIII inhibitor or a history of FVIII inhibitor
- Any number of FVIII exposure days, including PUPs
- BMI \<30
- Age ≥5 years to ≤45 years
- Medical documentation of bleeding events, outcomes and hemostatic product usage for 12 months prior to study enrollment
- Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the health-related questionnaires, activity tracking, and bleed diaries, using systems provided during the study
- Willingness to undergo a Stimate/DDAVP challenge (only if the subject reports no adverse event associated with prior Stimate \[DDAVP/desmopressin acetate\] use); Stimate/DDAVP challenge will not be performed if the patient has a documented history of lack of response as defined by an increase of FVIII \< 2 times baseline level
- Adequate hepatic function, defined as total bilirubin ≤1.5 × age-adapted upper limit of normal (ULN) (excluding Gilbert's syndrome) and both AST and ALT ≤3 × age-adapted ULN at the time of screening, and no clinical signs or known laboratory/radiographic evidence consistent with cirrhosis
- Adequate hematologic function, defined as a platelet count ≥100,000/μL and a PT≤1.5 times the ULN at the time of screening
- Adequate renal function, defined as serum creatinine ≤2.5 × age-adapted ULN and creatinine clearance ≥30 mL/min by Cockcroft-Gault formula
- Exclusion Criteria:
- Inherited or acquired bleeding disorder other than mild congenital hemophilia A (baseline FVIII level of \>5% to 30%)
- Any bleeding disorder other than or in addition to mild hemophilia A
- Current or prior inhibitor to FVIII (any titer)
- Female sex
- History of CVD, risk of CVD by the ASCVD risk estimator (defined as a subject having \>20% risk of a cardiovascular event within the next 10 years if the subject is ≥20 years of age) and/or a history of ischemic heart disease \[ICD codes 120-125\]
- High risk for TMA (eg, have a previous medical or family history of TMA), in the Study Investigator's judgment
- History of illicit drug or alcohol abuse by report or in the Study Investigator's judgment
- Previous (within the last 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
- Other conditions (eg, certain autoimmune diseases) that may currently increase the risk of bleeding or thrombosis
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the Hemlibra (emicizumab) injection
- Known HIV infection with CD4 counts \<200 cells/μL. HIV infection with CD4 counts ≥200 cells/μL permitted
- Use of systemic immunomodulators (eg, interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy
- Concomitant disease, condition, significant abnormality on screening evaluations or laboratory tests, or treatment that could interfere with the conduct of the study, or that would, in the opinion of the Study Investigator, pose an additional unacceptable risk in administering study drug to the patient
- Receipt of any of the following:
- Hemlibra (emicizumab) in a prior investigational study
- An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration
- A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter
- Any other investigational drug currently being administered or planned to be administered
- Inability to comply with the study protocol in the opinion of the Study Investigator
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.