A Clinical Study to Assess Sutacimig in Participants With Congenital Factor VII Deficiency

Hemab

Status and phase: Enrolling; Phase 2

Conditions: Congenital Factor VII Deficiency

Treatments: Drug: Sutacimig

Study type: Interventional

Funder types: Industry

Identifiers

NCT07347249

HMB-001-201

Details and patient eligibility

About

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).

Full 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%.

Enrollment: 18 estimated patients

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age 18 to 60 years, inclusive, at the time of signing informed consent.
2. Diagnosis of FVIID defined by Factor VII:C activity < 10% documented on ≥ 2 different laboratory measurements by local laboratory assessment.
3. 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.
4. Has the ability to provide informed consent to participate in the trial.

Exclusion criteria

1. Presence of known inhibitors to FVII or FVIIa
2. History of clinically significant hypersensitivity associated with monoclonal antibody therapies.
3. History of venous or arterial thrombosis or thromboembolic disease, with the exception of catheter-associated superficial vein thrombosis.
4. 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%.
5. Clinically significant comorbidity that may interfere with study participation.
6. 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])
7. Female participants who are pregnant or breastfeeding.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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18 participants in 2 patient groups

Participants with a FVII(a) level of < 10%

Experimental group

Treatment:

Drug: Sutacimig

Drug: Sutacimig

Participants with a FVII(a) level of ≥10%

Experimental group

Treatment:

Drug: Sutacimig

Drug: Sutacimig