Overview
Open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of a single dose of sutacimig monotherapy in participants with congenital FVII deficiency (FVIID).
Description
The objective is to administer a single dose of sutacimig and to evaluate safety, pharmacokinetics, and pharmacodynamics. Two cohorts may be evaluated. Cohort A is defined by participants with a FVII(a) level of \< 10%. Cohort B is defined by participants with a FVII(a) level of ≥10%.
Eligibility
Inclusion Criteria:
- Age 18 to 60 years, inclusive, at the time of signing informed consent.
- Diagnosis of FVIID defined by Factor VII:C activity \< 10% documented on ≥ 2 different laboratory measurements by local laboratory assessment.
- Severe bleeding history characterized by history of a major bleeding event and/or receipt of recombinant activated FVII or fresh frozen plasma as treatment for bleeding or a severe clinical bleeding history as defined by the Investigator.
- Has the ability to provide informed consent to participate in the trial.
Exclusion Criteria:
- Presence of known inhibitors to FVII or FVIIa
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies.
- History of venous or arterial thrombosis or thromboembolic disease, with the exception of catheter-associated superficial vein thrombosis.
- Known thrombophilia risk by the following criteria: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/Prothrombin gene mutation, antithrombin \<50%, congenital protein C, and protein S deficiency with levels \<50%.
- Clinically significant comorbidity that may interfere with study participation.
- Use of concomitant therapy not permitted during the study (i.e., other platelet inhibitors, desmopressin, fibrinolysis inhibitors, except if used as local treatment \[e.g., for oral bleeds\])
- Female participants who are pregnant or breastfeeding.