Phase 2
N=15
A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
Factor XIII Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT00883090 ↗Enrolled (actual)
15
Serious AEs
0.0%
Results posted
Jan 2012
Primary outcome: Primary: Peak FXIII Concentration at Steady State — 0.9 Units/mL
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- FXIII Concentrate (Human) (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- CSL Behring
- Primary completion
- Apr 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Peak FXIII Concentration at Steady State |
0.9 | — |
| PRIMARY Trough FXIII Concentration at Steady State |
0.05 | — |
| PRIMARY Time to Peak Concentration |
1.7 | — |
| PRIMARY Incremental Recovery |
0.02 | — |
| PRIMARY Terminal Half-life |
6.6 | — |
| PRIMARY Area Under the Curve at Steady State |
184.0 | — |
| PRIMARY Clearance |
0.25 | — |
| PRIMARY Volume of Distribution at Steady State |
51.1 | — |
| PRIMARY Mean Residence Time |
10.0 | — |
| SECONDARY Adverse Events |
8 | — |
| SECONDARY Laboratory Safety Parameters |
— | — |
| SECONDARY Vital Signs |
— | — |
Summary
Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.
Eligibility Criteria
Inclusion Criteria
- Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
- Documented congenital FXIII deficiency that requires prophylactic treatment with a FXIII containing product.
- Males and females of any age with congenital FXIII deficiency.
- Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive
Exclusion Criteria
- Diagnosis of acquired FXIII deficiency
- Administration of a FXIII-containing product, including blood transfusions or other blood products within 4 weeks prior to the planned Day 0
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Known or suspected to have antibodies towards FXIII
- Use of any other investigational medicinal product within 4 weeks prior to the Baseline Visit (Day 0)
- Positive result at screening for human immunodeficiency virus (HIV)
- Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal
- Fibrinogen < lower limit of normal
- Active bleeding
- Pregnant or breast-feeding
- Intention to become pregnant during the course of the study
- Female subjects of childbearing potential not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study
- Surgical procedure anticipated during the study period
- Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance
Data sourced from ClinicalTrials.gov (NCT00883090). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.