Safety and Pharmacokinetics of Recombinant Factor XIII in Patients With Congenital Factor Xlll Deficiency

Study Overview
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Brief Summary
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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.

Official Title
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A Phase 1 Escalating Dose Study of the Safety and Pharmacokinetics of Recombinant Factor XIII in Patients With Congenital Factor XIII Deficiency

Conditions
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Congenital Bleeding Disorder, Congenital FXIII Deficiency

Intervention / Treatment
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* Drug: catridecacog


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rFXIII<br> | Drug: catridecacog<br>* 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).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse events |  | Days 0-28 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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 |