Congenital Factor VII Deficiency
Conditions
Keywords
Congenital Factor VII Deficiency, Factor VII Deficiency, FVIID, Congenital coagulation factor VII (FVII) deficiency
Brief summary
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
DRUGSutacimig
Sutacimig is a subcutaneously administered, bispecific antibody being developed as a prophylactic treatment option for congenital bleeding disorders.
Sponsors
Hemab ApS
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) | Day 1 through Day 57 |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetic Parameter: Maximum observed plasma concentration (Cmax) of sutacimig | Day 1 through Day 57 |
| Pharmacokinetic Parameter: Time to reach maximum observed plasma concentration (Tmax) | Baseline through Day 57 |
| Pharmacokinetic Parameter: Area under the plasma concentration-time curve from time zero to last quantifiable concentration (AUClast) | Day 1 through Day 57 |
| Pharmacokinetic Parameter: Area under the curve from time zero to extrapolated infinite time (AUCinf) | Day 1 through Day 57 |
| Pharmacokinetic Parameter: Terminal elimination phase half-life (T1/2) | Day 1 through Day 57 |
| Pharmacodynamic Parameter: Total Factor VII | Day 1 through Day 57 |
| Pharmacodynamic Parameter: Factor VII Activity | Day 1 through Day 57 |
| Pharmacodynamic Parameter: Prothrombin time (PT) Measurement | Day 1 through Day 57 |
| Pharmacodynamic Parameter: Activated partial thromboplastin time (aPTT) Measurement | Day 1 through Day 57 |
| Anti-drug antibody levels | Day 1 through Day 57 |
Countries
United Kingdom
Contacts
CONTACTHemab Aps
Outcome results
None listed