Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Standard of Care FIX Replacement therapy · 1 trial · 2 indications
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study multiplication(\*)365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per efficacy analysis set was reported. Abbreviations used: AAV6 = adeno-associated virus 6; AAV-Spark100 = Bioengineered AAV capsid, derived from a naturally occurring AAV serotype; nAb = neutralizing antibodies.
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per protocol analysis set was reported.
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per protocol amendment 5 analysis set was reported.
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective data collection period in hemophilia B participants per protocol amendment 5 analysis set was reported.
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective and prospective data collection period in hemophilia B participants per protocol amendment 5 analysis set was reported.
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia A participants per efficacy analysis set was reported.
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia A participants per protocol analysis set was reported.
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia A participants per protocol amendment 5 analysis set was reported.
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective data collection period in hemophilia A participants per protocol amendment 5 analysis set was reported.
ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective and prospective data collection period in hemophilia A participants per protocol amendment 5 analysis set was reported.
| Arm | Type | Description |
|---|---|---|
| Standard of Care FIX replacement therapy | OTHER | - |
| Standard of Care FVIII replacement therapy | OTHER | - |
| Name | Type | Description |
|---|---|---|
| Standard of Care FIX Replacement therapy | DRUG | There is no investigational product being administered. Subjects will be administering their own standard of care FIX replacement therapy. |
| Standard of Care FVIII Replacement therapy | DRUG | There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy. |
Inclusion Criteria: Hemophilia B Population: 1. Evidence of a signed and dated informed consent document indicating that the participant has been informed of all pertinent aspects of the study. 2. Willing and able to comply with scheduled visits, FIX prophylaxis treatment plan, laboratory tests an...
Standard of Care FIX Replacement therapy is used for hemophilia B, a bleeding disorder caused by a deficiency of clotting factor IX. It is an investigational replacement therapy being studied in adult patients with hemophilia B and factor IX levels of 2% or less. The therapy is intended to provide the missing clotting factor to help prevent bleeding episodes.
Standard of Care FIX Replacement therapy is being developed by Pfizer, Inc., a biopharmaceutical company traded on the New York Stock Exchange under the ticker symbol PFE. Pfizer is conducting clinical research to evaluate the efficacy and safety of this factor IX replacement therapy in patients with hemophilia B.
Standard of Care FIX Replacement therapy is in Phase 3 clinical development. It is an investigational therapy, meaning it has not yet been approved by regulatory authorities. The Phase 3 study has been completed, and the therapy remains under evaluation for its efficacy and safety in adult patients with hemophilia B.
Standard of Care FIX Replacement therapy was studied in a Phase 3 clinical trial registered as NCT03587116. This completed study evaluated prospective efficacy and safety data of current factor IX prophylaxis replacement therapy in adult hemophilia B subjects with factor IX levels of 2% or less. The trial enrolled 212 male participants aged 18 years and older across multiple countries.
Standard of Care FIX Replacement therapy is a factor IX replacement therapy. The term FIX refers to clotting factor IX, and the therapy is designed to replace this deficient clotting factor in patients with hemophilia B. It is administered as prophylaxis to prevent bleeding episodes in adult patients with severe or moderately severe hemophilia B.