A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00885742
• Date of registration: 21/04/2009
• Prospective Registration: Yes
• Primary sponsor: CSL Behring
• Public title: A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
• Scientific title: A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
• Date of first enrolment: August 2009
• Target sample size: 41
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00885742
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Spain, United States

Contacts

• Name: Program Director, Clinical R&D
• Affiliation: CSL Behring

Key inclusion & exclusion criteria

Inclusion Criteria:

- Written informed consent/assent for study participation obtained before undergoing any
study-specific procedures

- Documented congenital FXIII deficiency which 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)

- Known Positivity for human immunodeficiency virus (HIV) or a positive result for HIV
at the Screening Visit of this study or the FXIII study 2002 (NCT00883090).

- 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 Factor XIII Study BI71023_2002 (NCT00883090)

- Fibrinogen level less than 85% of the lower limit of normal at the Screening Visit of
this study or the Factor XIII Study BI71023_2002 (NCT00883090)

- Active bleeding = Common Terminology Criteria for Adverse Events (CTCAE) Grade 2
and/or = moderate between the Screening and Baseline Visits

- 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

- Suspected inability (e.g., language problems) or unwillingness to comply with study
procedures or history of noncompliance



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Factor XIII Deficiency

Intervention(s)

Biological: FXIII Concentrate (Human)

Primary Outcome(s)

The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event) [Time Frame: Up to week 52]

Secondary Outcome(s)

Trough FXIII Concentration at Steady State [Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion.]

Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels [Time Frame: 12 months]

Achievement of Trough Factor XIII Levels of 5% or Higher. [Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion.]

Peak FXIII Concentration at Steady State [Time Frame: At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.]

Adverse Events [Time Frame: 12 months]

Time to Peak Concentration [Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.]

Incremental Recovery [Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.]

Secondary ID(s)

2009-010722-19

BI71023_3001

1482

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 04/07/2012
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00885742