Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
Study Details
- Hemophilia A
- Biological: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
- Main inclusion Criteria
- Males who have been followed on routine Factor VIII prophylaxis therapy during the lead-in study (C0371004) and have \> = 150 documented exposure days to a Factor VIII protein product
- Moderately severe to severe hemophilia A (Factor VIII activity \< =1%)
- Suspension of FVIII prophylaxis therapy post study drug infusion
- Main exclusion Criteria
- Anti-AAV6 neutralizing antibodies
- History of inhibitor to Factor VIII
- Laboratory values at screening visit that are abnormal or outside acceptable study limits
- Significant and/or unstable liver disease, biliary disease, significant liver fibrosis
- Conditions associated with increased thromboembolic risk such as inherited or acquired thrombophilia, or a history of thrombotic events
- Planned surgical procedure requiring Factor VIII surgical prophylactic factor treatment 12 months from screening visit
- Active hepatitis B or C
- Serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 and/or viral load \>20 copies/mL
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.