Phase 3
N=61
An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Factor XIII Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT00945906 ↗Enrolled (actual)
61
Serious AEs
3.3%
Results posted
Oct 2012
Primary outcome: Primary: Adverse Events — 42; 2; 2 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- FXIII Concentrate (Human) (FXIII) (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- CSL Behring
- Primary completion
- Aug 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Adverse Events |
42; 2; 2 | — |
| SECONDARY Hematology and Chemistry Testing |
1; 1 | — |
| SECONDARY FXIII Antibody Testing |
1; 60 | — |
| SECONDARY FXIII Concentration |
0.0987; 0.1177; 0.1238; 0.1075; 0.1025; 0.1160 | — |
| SECONDARY Number of Subjects With at Least One Bleeding Episode |
10; 1 | — |
| SECONDARY Number of Bleeding Episodes |
14 | — |
Summary
Congenital deficiency of factor XIII is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII. Factor XIII Concentrate works by assisting blood in the usual clotting process, thereby preventing bleeding.
In this study, patients will be treated with FXIII Concentrate (Human) and followed closely to determine that they receive the dose of FXIII Concentrate (Human) that will best minimize the chance of bruising and bleeding. The purpose of the study is to provide FXIII Concentrate (Human) to patients until the product becomes commercially available in the United States.
Eligibility Criteria
Inclusion Criteria
- Written informed consent/assent for study participation obtained before undergoing any study specific procedures
- Diagnosed with congenital FXIII deficiency requiring prophylactic treatment
- Males and females of any age
Exclusion Criteria
- Diagnosis of acquired FXIII deficiency
- Administration of a FXIII-containing product, including blood transfusions or other blood products, within 3 weeks prior to the Baseline/Day 0 Visit
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Use of any other IMP within 4 weeks prior to Baseline/Day 0 Visit
- 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
- Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance
- Any laboratory finding or medical condition which, in the opinion of the Investigator, would put the subject or subject's disease management at risk
Data sourced from ClinicalTrials.gov (NCT00945906). 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.