Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Testing of hepatic AAV Vector integration · 1 trial · 2 indications
FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL).
Factor activity level will be reported. Factor levels may be measured using different assay methods including a one-stage assay or by chromogenic substrate assay and a second one-stage assay.
| Arm | Type | Description |
|---|---|---|
| Hemophilia A / giroctocogene fitelparvovec | OTHER | Participants have received treatment with giroctocogene fitelparvovec in a previous study and are not receiving any investigational product in this study |
| Hemophilia B / fidanacogene elaparvovec | OTHER | Participants have received treatment with fidanacogene elaparvovec in a previous study and are not receiving any investigational product in this study |
| Name | Type | Description |
|---|---|---|
| Testing of hepatic AAV Vector integration | DIAGNOSTIC_TEST | Evaluation of AAV vector integration in participants for whom a sample of liver has been obtained through biopsy or surgical resection when clinically indicated |
Inclusion Criteria: -Only participants who received investigational giroctocogene fitelparvovec or fidanacogene eleparvovec and were enrolled in a Pfizer-sponsored study (C0371002, C0371003, C0371005, C3731001, C3731003) are eligible. Exclusion Criteria: -None
Testing of hepatic AAV Vector integration is being studied for use in Hemophilia A, a bleeding disorder. It is part of a Phase 3 clinical trial that also includes patients with Hemophilia B. The drug is being evaluated for safety and effectiveness in this patient population.
Testing of hepatic AAV Vector integration is being developed by Pfizer, Inc., which trades under the ticker symbol PFE on the stock exchange. The company is conducting a Phase 3 clinical trial to evaluate the drug's safety and effectiveness in patients with Hemophilia A.
Testing of hepatic AAV Vector integration is currently in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 3 trial is recruiting participants to assess the drug's safety and effectiveness.
Testing of hepatic AAV Vector integration is being studied in a Phase 3 clinical trial with the identifier NCT05568719. This trial is recruiting 173 male participants aged 18 years and older with Hemophilia A or Hemophilia B. The study is being conducted in the United States, Australia, Canada, South Korea, Sweden, Taiwan, and Turkey.
Testing of hepatic AAV Vector integration is not the same as Giroctocogene Fitelparvovec. The clinical trial NCT05568719 is evaluating both Giroctocogene Fitelparvovec for Hemophilia A and Fidanacogene Elaparvovec for Hemophilia B. Testing of hepatic AAV Vector integration is a separate investigational drug being studied in the same trial.