A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- CSL Behring
- Enrollment
- 61
- Locations
- 27
- Primary Endpoint
- Adverse Events
Study Overview
Brief 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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Masking
- None
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Outcomes
Primary Outcomes
Adverse Events
Time Frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Number of subjects with any treatment-emergent adverse event (AE), treatment-related AE or serious AE (SAE). Treatment-related AEs are defined as AEs whose relationship to treatment is related, or possibly related and AEs with missing relationship.
Secondary Outcomes
- Hematology and Chemistry Testing(After the first infusion and at the end-of-study (or withdrawal) visit.)
- FXIII Antibody Testing(Before the first infusion, then every 48 weeks, at the end-of-study (or withdrawal) visit and after a bleeding episode requiring treatment with a Factor XIII -containing product.)
- FXIII Concentration(Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.)
- Number of Subjects With at Least One Bleeding Episode(After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.)
- Number of Bleeding Episodes(After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.)