A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects with Congenital Factor XIII Deficiency Estudio 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

• Register: EUCTR
• Last refreshed on: 26 June 2012
• Main ID: EUCTR2009-010722-19-ES
• Date of registration: 11/08/2009
• Prospective Registration: Yes
• Primary sponsor: CSL Behring LLC
• Public title: A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects with Congenital Factor XIII Deficiency Estudio 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
• Scientific title: A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects with Congenital Factor XIII Deficiency Estudio 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
• Date of first enrolment: 04/11/2009
• Target sample size: 40
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-010722-19
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other:

Countries of recruitment

Spain

Key inclusion & exclusion criteria

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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Congenital Factor XIII deficiency Deficiencia congénita de factor XIII
MedDRA version: 9.1 Level: LLT Classification code 10016083 Term: Factor XIII deficiency

Intervention(s)

Trade Name: Fibrogammin® P
Product Name: Factor XIII Concentrate (Human), Pasteurized
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Factor XIII Concentrate (Human), Pasteurized
Concentration unit: U unit(s)
Concentration type: equal
Concentration number: 1250-

Primary Outcome(s)

Secondary Objective: Secondary objectives of this study are as follows:
? To evaluate the association between FXIII activity trough levels and clinical efficacy
? To collect and evaluate additional long-term pharmacokinetic (PK) data in this
population
? To collect and evaluate and long-term safety data in this population.
? To evaluate hemostatic efficacy in treatment of acute bleeding events and for
surgery prophylaxis

Main Objective: The primary objective of this study is to collect and evaluate observational long term efficacy data with regard to the frequency and severity of bleeding episodes in this population.

Primary end point(s): The primary efficacy endpoints in this study are frequency and severity of bleeding episodes following prophylactic treatment. Long-term PK data, safety data, and the assessment of hemostatic efficacy (following FXIII concentrate treatment of acute bleeding events and following scheduled surgery where FXIII concentrate was used for prophylaxis prior to the scheduled surgery) are also endpoints in this study.

Secondary ID(s)

BI71023_3001