Congenital Bleeding Disorder, Congenital FXIII Deficiency
Conditions
Brief summary
This trial was conducted in the United States of America (USA). The aim of this trial was to investigate safety and pharmacokinetics of escalating single doses of catridecacog (recombinant factor XIII, rFXIII) in patients with congenital factor XIII deficiency.
Interventions
DRUGcatridecacog
Single doses of rFXIII administered intravenously (IV) to two subjects in each of the five dose levels (2, 6, 20, 50 and 75 U/kg).
Sponsors
Novo Nordisk A/S
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Documental congenital FXIII deficiency * Normal platelet count and clotting parameters * Adequate renal and hepatic function * If female and of child-bearing potential, negative serum pregnancy test within 7 days of enrollment * If a sexually active male or a sexually active female of child-bearing potential, agreement to use a medically accepted form of contraception from the time of enrollment to completion of all follow-up study visits * Negative drug and alcohol screens
Exclusion criteria
* Received blood products or FXIII concentrates within 4 weeks of study enrollment * Known antibodies to FXIII * Hereditary or acquired coagulation disorder other than FXIII deficiency * Previous history of autoimmune disorders involving autoantibodies e.g., systemic lupus erythematosus * Previous history of thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis or administration of any antithrombotic or antiplatelet drugs within 7 days of study enrollment * Received treatment with any experimental agent within 30 days of study enrollment * Any surgical procedure in the 30 days prior to enrollment * Donated blood within 30 days prior to enrollment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse events | Days 0-28 |
Secondary
| Measure | Time frame |
|---|---|
| Incidence of clinically significant changes from baseline in physical examination or laboratory measurements | Days 0-28 |
| Incidence of rFXIII antibodies, as measured by ELISA (Enzyme-Linked Immuno Sorbent Assay) | Days 0-28 |
| Incidence of yeast antibodies | Days 0-28 |
| FXIII activity measured by the Berichrom® assay | Days 0-28 |
Countries
United States
Outcome results
None listed