A Clinical Study to Assess the Safety & Efficacy of Sutacimig in Participants with Congenital FVIID

• Research type

  Research Study

• Full title

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

• IRAS ID

  1012417

• Contact name

  Kristen Pappas

• Contact email

  kristen@hemab.com

• Sponsor organisation

  Hemab ApS

• Research summary

  Congenital factor VII (FVII) deficiency is a rare bleeding disorder caused by genetic mutations that lead to low levels or poor function of FVII. When an injury occurs, FVII normally works with tissue factor to help start blood clotting. A lack of FVIIa results in less thrombin, which is necessary for proper blood clotting, leading to bleeding episodes.
  Sutacimig (HMB-001) is a new subcutaneous treatment developed by Hemab. It binds to FVIIa and activated platelets to help generate thrombin, similar to recombinant activated FVII (rFVIIa).
  This Phase 2b study will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of a single dose of sutacimig in participants with FVIID who have a history of bleeding. Participants will be divided into two cohorts: Cohort A with FVII(a) levels <10% and Cohort B with levels ≥10%.
  Dosing will begin with three participants in Cohort A at Dose Level 1 (0.1 mg/kg). After enrolling three participants at each dose level, the Safety Review Committee (SRC) will review data through Day 29 and may recommend dose escalation, expansion, or other actions. Cumulative data across all cohorts will be reviewed on an ongoing basis to determine if stopping rules have been met. If at any time during enrollment, data suggest that stopping rules may have been met, then dosing will be suspended to permit formal review of available data by the SRC. Participants will be dosed at the clinic on Day 1 and will remain at the site for a minimum of 4 hours post-dose for observation and to complete study assessments. Participants will return to the clinic at the time points noted in the Schedule of Assessments. The planned duration of study participation is approximately 4 months. This includes a Screening Period of up to 60 days, a treatment period of 1 day, and a follow-up period of 56 days. This is single site study. Up to 18 participants are expected to be enrolled in the study.

• REC name

  London - Fulham Research Ethics Committee

• REC reference

  25/LO/0547

• Date of REC Opinion

  20 Aug 2025

• REC opinion

  Further Information Favourable Opinion