A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects with Congenital Factor XIII DeficiencyEstudio prospectivo, multicéntrico, con etiqueta abierta, en fase 3b, del concentrado de factor XIII derivado del plasma humano en sujetos con deficiencia congénita de factor XIII

• Conditions: MedDRA version: 9.1Level: LLTClassification code 10016083Term: Factor XIII deficiency; Congenital Factor XIII deficiencyDeficiencia congénita de factor XIII

• Registration Number: EUCTR2009-010722-19-ES
• Lead Sponsor: CSL Behring LLC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-08-11
• Last Update Posted: 26 June 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1. Written informed consent/assent for study participation obtained before
undergoing any study-specific procedures.
2. Diagnosed with severe congenital FXIII deficiency (< 10 U/dL at time of initial
diagnosis).
3. Males and females of any age with congenital FXIII deficiency.
4. Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Diagnosis of acquired FXIII deficiency.
2. Administration of a FXIII-containing product, including blood transfusions or other
blood products within 4 weeks prior to the planned Day 0.
3. Any known congenital or acquired coagulation disorder other than congenital
FXIII deficiency.
4. Known or suspected to have antibodies towards FXIII.
5. Use of any other investigational medicinal product within 4 weeks prior to the
Baseline Visit (Day 0).
6. Positive result for human immunodeficiency virus (HIV).
7. Serum aspartate transaminase (AST) or serum alanine transaminase (ALT)
concentration > 2.5 times the upper limit of normal at the Screening Visit of this
study or at the Day 56 Visit of FXIII Study 2002.
8. Fibrinogen level below the lower limit of normal at the Screening Visit of this
study or the FXIII Study 2002.
9. Active bleeding ? CTCAE Grade 2 and/or ? moderate (as defined in Section 8.2)
between the Screening and Baseline Visits.
10. Pregnant or breast-feeding.
11. Intention to become pregnant during the course of the study.
12. 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.
13. Suspected inability (e.g., language problems) or unwillingness to comply with
study procedures or history of noncompliance.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified