A Clinical Study to Assess Sutacimig in Participants With Congenital Factor VII Deficiency
ConditionCongenital Factor VII Deficiency
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-60
SponsorHemab ApS
About this trial
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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%.
Trial design
Design model
Sequential
Treatments tested in this trial
Sutacimig
DrugSutacimig is a subcutaneously administered, bispecific antibody being developed as a prophylactic treatment option for congenital bleeding disorders.
Sutacimig
DrugSutacimig is a subcutaneously administered, bispecific antibody being developed as a prophylactic treatment option for congenital bleeding disorders.
Treatment groups
18 Participants
are divided into 2 treatment groupsGroup A: Participants with a FVII(a) level of < 10%Experimental treatment 1 intervention
Group B: Participants with a FVII(a) level of ≥10%Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence of treatment-emergent adverse events (TEAEs)
Time frame
Day 1 through Day 57Secondary outcomes
1
Pharmacokinetic Parameter: Maximum observed plasma concentration (Cmax) of sutacimig
Time frame
Day 1 through Day 572
Pharmacokinetic Parameter: Time to reach maximum observed plasma concentration (Tmax)
Time frame
Baseline through Day 573
Pharmacokinetic Parameter: Area under the plasma concentration-time curve from time zero to last quantifiable concentration (AUClast)
Time frame
Day 1 through Day 574
Pharmacokinetic Parameter: Area under the curve from time zero to extrapolated infinite time (AUCinf)
Time frame
Day 1 through Day 57 Source ClinicalTrials.gov