A Clinical Study to Assess Sutacimig in Participants With Congenital Factor VII Deficiency
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Sutacimig, Sutacimig.
- Who it may be relevant to
- Registry conditions: Congenital Factor VII Deficiency. Basic parameters: 18 years — 60 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Clinical Study to Assess the Safety and Efficacy of Sutacimig in Participants With Congenital Factor VII Deficiency
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).
Detailed 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%.
Interventions
- Drug Sutacimig
Sutacimig is a subcutaneously administered, bispecific antibody being developed as a prophylactic treatment option for congenital bleeding disorders. - Drug Sutacimig
Sutacimig is a subcutaneously administered, bispecific antibody being developed as a prophylactic treatment option for congenital bleeding disorders.
Primary outcome measures
- Incidence of treatment-emergent adverse events (TEAEs) [Time frame: Day 1 through Day 57]
Secondary outcome measures (10)
- Pharmacokinetic Parameter: Maximum observed plasma concentration (Cmax) of sutacimig [Time frame: Day 1 through Day 57]
- Pharmacokinetic Parameter: Time to reach maximum observed plasma concentration (Tmax) [Time frame: Baseline through Day 57]
- Pharmacokinetic Parameter: Area under the plasma concentration-time curve from time zero to last quantifiable concentration (AUClast) [Time frame: Day 1 through Day 57]
- Pharmacokinetic Parameter: Area under the curve from time zero to extrapolated infinite time (AUCinf) [Time frame: Day 1 through Day 57]
- Pharmacokinetic Parameter: Terminal elimination phase half-life (T1/2) [Time frame: Day 1 through Day 57]
- Pharmacodynamic Parameter: Total Factor VII [Time frame: Day 1 through Day 57]
- Pharmacodynamic Parameter: Factor VII Activity [Time frame: Day 1 through Day 57]
- Pharmacodynamic Parameter: Prothrombin time (PT) Measurement [Time frame: Day 1 through Day 57]
- Pharmacodynamic Parameter: Activated partial thromboplastin time (aPTT) Measurement [Time frame: Day 1 through Day 57]
- Anti-drug antibody levels [Time frame: Day 1 through Day 57]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United Kingdom · 1 center
- Royal London Hospital — London
Identifiers
NCT: NCT07347249 · HMB-001-201