A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
Study Details
- Hemophilia B
- Previous experience with FIX therapy (≥50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product) with a current stable prophylaxis regimen for \>2 months prior to enrollment and intention to use FIX replacement therapy for the duration of the study
- No known hypersensitivity to FIX replacement product
- Willing to be contacted about a potential future clustered regularly interspaced short palindromic repeats (CRISPR)-based Factor 9 (F9) gene insertion clinical trial in which they may have the opportunity to screen for enrollment
- History of any coagulation disorder; requires anticoagulant therapy
- Lack of adherence with documentation of bleeds and/or prophylaxis replacement therapy administration in the opinion of the investigator, based on medical history
- History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions, as defined in the protocol
- Bethesda inhibitor titer greater than the upper limit of normal (ULN) at screening
- Any detectable pre-existing antibodies to the Adeno-associated virus serotype 8 (AAV8) capsid; as measured by an assay at prescreening, as defined in the protocol
- Is positive for hepatitis B or C at screening, as defined in protocol
- If any of the following pre-existing diagnoses are documented:
- Cholestatic liver disease
- Liver cirrhosis
- Portal hypertension; or
- Splenomegaly; or
- Hepatic encephalopathy
- History of arterial or venous thrombo-embolic events, as defined in the protocol
- History of clinically significant cardiovascular, respiratory, hepatic, renal (including nephrotic syndrome), gastrointestinal (including protein-losing enteropathy), endocrine, hematological (including thrombophilia), psychiatric, or neurological disease, as assessed by the investigator that may confound the results of the study or poses an additional risk to the participant by study participation
- Previously received of any AAV-gene based therapy with a marketed gene therapy or in a clinical trial or intent to receive approved or investigational AAV-gene based therapy during the study period
- NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply
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.