Clinical Trial of NXT007 for the Prophylactic Treatment of Hemophilia A in Infants and Children
Study Details
- Hemophilia A
- Drug: NXT007
- Age \<12 years at the time of signing Informed Consent Form; or age ≥12 and \<18 years with body weight \<40 kg
- Diagnosis of severe (Factor VIII coagulation protein activity \[FVIII:C\] \<1 International Unit per decilitre \[IU/dL\]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A (HA) with or without inhibitors against factor VIII (FVIII)
- For potential participants with moderate HA without inhibitors, fulfillment of at least one of the following criteria: Current prophylaxis with long-term prophylaxis intention due to a severe bleed phenotype; For those not treated with prophylaxis: at least one traumatic joint or critical muscle bleed in the last 6 months or ≥2 spontaneous bleeds/year or 5 bleeds/year, including traumatic bleeds; Signs of joint degeneration compatible with hemophilic arthropathy (e.g., subchondral bone changes or the presence of synovitis) as assessed by any imaging assessment (e.g., ultrasound, MRI, radiograph); Bleeding at a critical site (e.g., intracranial).
- Diagnosis of mild (FVIII:C between \>5 IU/dL and \<40 IU/dL) congenital hemophilia A with chronic FVIII inhibitors, defined as documented FVIII inhibitor (≥0.6 BU/mL or ≥1.0 BU/mL only for laboratories with a historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and chronic reduction of endogenous baseline FVIII:C to \<5 IU/dL for ≥12 months
- Documentation of the details of prophylactic and episodic FVIII treatment, bypassing agent (BPA) treatment, emicizumab prophylaxis treatment, and the number and type of bleeding episodes for at least the last 6 months prior to screening if appropriate
- For potential participants taking on-demand treatments prior to study entry: agreement to move to a prophylaxis treatment with NXT007
- Adequate renal, hepatic, and hematologic function, as defined in the protocol
- Sensitivity to any of the study investigations, or components thereof, or drug or other allergy that, in the opinion of the investigator, contraindicates participation in the study
- Use of systemic immunomodulators (e.g., interferon or rituximab) at the time of enrollment or planned use during the study, except for antiretroviral therapy to treat HIV
- Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario
- History or conditions, other than HA, which may indicate hypo- or hypercoagulopathy risk
- Planned surgery (excluding minor procedures, such as non-molar tooth extraction or incision and drainage) during the study
- History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy)
- Any serious medical condition or abnormality in clinical laboratory tests that precludes an individual's safe participation in and completion of 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.