Clinical trial NCT00056589: Safety and Pharmacokinetics of Recombinant Factor XIII in Patients With Congenital Factor Xlll Deficiency
Official title: A Phase 1 Escalating Dose Study of the Safety and Pharmacokinetics of Recombinant Factor XIII in Patients With Congenital Factor XIII Deficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Congenital Bleeding Disorder
Congenital FXIII Deficiency
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2003-03-20
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of adverse events
timeFrame: Days 0-28
Secondary outcomes:
measure: Incidence of clinically significant changes from baseline in physical examination or laboratory measurements
timeFrame: Days 0-28
measure: Incidence of rFXIII antibodies, as measured by ELISA (Enzyme-Linked Immuno Sorbent Assay)
timeFrame: Days 0-28
measure: Incidence of yeast antibodies
timeFrame: Days 0-28
measure: FXIII activity measured by the Berichrom® assay
timeFrame: Days 0-28
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Novo Nordisk Investigational Site
state: California
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Lovejoy AE, Reynolds TC, Visich JE, Butine MD, Young G, Belvedere MA, Blain RC, Pederson SM, Ishak LM, Nugent DJ. Safety and pharmacokinetics of recombinant factor XIII-A2 administration in patients with congenital factor XIII deficiency. Blood. 2006 Jul 1;108(1):57-62. doi: 10.1182/blood-2005-02-0788. Epub 2006 Mar 23.