Sutacimig for adults with congenital Factor VII deficiency

A Clinical Study to Assess the Safety and Efficacy of Sutacimig in Participants With Congenital Factor VII Deficiency

This will test a single dose of sutacimig in adults with congenital Factor VII deficiency to see if it is safe and improves clotting measures.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Ages: 18 Years to 60 Years
• Sex: All
• Sponsor: Hemab ApS (Industry-sponsored)
• Locations: 1 site (London)
• Trial ID: NCT07347249 on ClinicalTrials.gov

What this trial studies

This open-label Phase 2 trial gives a single dose of sutacimig as monotherapy to adults with congenital Factor VII deficiency and follows them for safety, tolerability, pharmacokinetics, pharmacodynamics, and signs of efficacy. Participants are separated into two cohorts based on FVII(a) activity (<10% versus ≥10%). Key data collected will include adverse events, drug levels, biomarker changes related to coagulation, and any bleeding outcomes. The study enrolls adults aged 18–60 with documented low FVII activity and a history of clinically significant bleeding.

Who should consider this trial

Good fit: Ideal candidates are adults 18–60 with documented congenital Factor VII deficiency (Factor VII:C <10% on at least two tests) and a history of major or clinically significant bleeding who can give informed consent.

Not a fit: Patients with known inhibitors to FVII/FVIIa, prior clinically significant thrombosis, certain thrombophilia genotypes, or a history of hypersensitivity to monoclonal antibodies are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If successful, sutacimig could offer a new treatment option that reduces bleeding risk or improves clotting function for people with severe congenital FVII deficiency.

How similar studies have performed: Replacement therapy with recombinant activated FVII has been used successfully for bleeding in FVII deficiency, but sutacimig represents a newer mechanism that is novel and not yet proven in this condition.

Eligibility criteria

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.

Where this trial is running

London

• Royal London Hospital — London, United Kingdom (Recruiting)

Study contacts

• Study coordinator: Hemab Aps

Conditions Congenital Factor VII Deficiency; Factor VII Deficiency; FVIID; Congenital coagulation factor VII deficiency