Congenital Coagulation Factor VII Deficiency Clinical Trial
Official title:
Multi-center, Randomized, Open-label, Parallel-Arm, Single-dose, Pharmacokinetic Study of rVIIa-FP (CSL689) in Subjects With Congenital Factor VII Deficiency
| Verified date | November 2016 |
| Source | CSL Behring |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
The purpose of this study is to investigate the pharmacokinetics (PK) and safety of rVIIa-FP (CSL689) in a total of 10 to 16 male or female adults with inherited coagulation factor VII (FVII) deficiency. Subjects will receive a single dose of their routine FVII replacement product (ie, either recombinant activated coagulation FVII [rFVIIa, eptacog alfa (activated)] or plasma-derived FVII [pdFVII]) as a comparator, and will then be randomly assigned to a single low dose or a single high dose of the study product CSL689 (8 subjects per CSL689 dose level). Serial blood samples for PK analysis will be taken up to 24 hours after the eptacog alfa (activated) or pdFVII injection, and up to 48 hours after the CSL689 injection. Subject safety will be routinely monitored throughout the study.
| Status | Completed |
| Enrollment | 9 |
| Est. completion date | October 2016 |
| Est. primary completion date | July 2016 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility |
Inclusion Criteria: - Proven congenital FVII deficiency. - Age = 18 years. - FVII level < 2% of normal levels. - Minimum of 50 previous exposure days to pdFVII (including prothrombin complex concentrates [PCCs]) or rFVIIa. Exclusion Criteria: - History of, or risk factors for, thromboembolic events, including known deep vein thrombosis. - Inhibitor to FVII or rFVIIa, current or historic. - Known or suspected hypersensitivity to hamster protein, to CSL689, or to any excipient of CSL689. - Known or suspected allergy to rFVIIa or hamster protein. - Major surgery within 1 month before screening. - Advanced atherosclerotic disease (ie, known history of ischemic heart disease, or ischemic stroke). - Human immunodeficiency virus (HIV)-positive subjects with cluster of differentiation 4 (CD4)+ lymphocyte count of < 200/µL at screening. - Use of an investigational agent within 30 days before the study. - Use of concomitant therapy not permitted during the study (ie, other platelet inhibitors, desmopressin, fibrinolysis inhibitors, except if used as local treatment [eg, for oral bleeds]) |
| Country | Name | City | State |
|---|---|---|---|
| Netherlands | Site Reference 5280023 | Njmegen | |
| Norway | Site Reference # 5780001 | Oslo |
| Lead Sponsor | Collaborator |
|---|---|
| CSL Behring |
Netherlands, Norway,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Terminal half-life of plasma FVIIa activity | Up to 48 hours after CSL689 injection | ||
| Primary | Maximum observed plasma FVIIa activity | Before injection and at up to 9 time points until 48 hours after injection | ||
| Primary | Area under the curve (AUC0-t) | Area under plasma FVIIa activity versus time curve from time 0 to last sample with quantifiable activity | Before injection and at up to 9 time points until 48 hours after injection | |
| Secondary | Total clearance | Total clearance of plasma FVIIa activity | Before injection and at up to 9 time points until 48 hours after injection | |
| Secondary | Volume of distribution of the terminal phase | Before injection and at up to 9 time points until 48 hours after injection | ||
| Secondary | AUC(0-inf) | Area under plasma FVIIa activity versus time curve from time 0 extrapolated to infinity | Before injection and at up to 9 time points until 48 hours after injection | |
| Secondary | Incremental recovery | Incremental recovery of plasma FVIIa activity | Before injection and at up to 9 time points until 48 hours after injection | |
| Secondary | Time of occurrence of maximum observed plasma FVIIa activity | Before injection and at up to 9 time points until 48 hours after injection | ||
| Secondary | Number of subjects with antibodies against Chinese hamster ovary protein and FVII | Up to 30 days after CSL689 injection | ||
| Secondary | Number of subjects with inhibitors against FVII | Up to 30 days after CSL689 injection |