Phase 3
N=41
Evaluation of Recombinant Factor XIII for Prevention of Bleeding in Patients With FXIII Inherited Deficiency
Congenital Bleeding Disorder · Congenital FXIII Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT00713648 ↗Enrolled (actual)
41
Serious AEs
14.6%
Results posted
Jul 2014
Primary outcome: Primary: Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period — 0.138 bleeding episodes per subject per year
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- catridecacog (Drug)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- All
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Apr 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period |
0.138 | — |
| SECONDARY Percentage of Subjects Having a Normal Clot Solubility One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits |
98.2; 91.3 | — |
| SECONDARY Level of FXIII Activity One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits |
0.782; 0.189 | — |
| SECONDARY Number of Subjects With rFXIII Antibody Development |
4 | — |
Summary
The trial is conducted in Europe, North America and Asia. The aim of this trial is to evaluate catridecacog (recombinant factor XIII (rFXIII)) treatment in patients with inherited FXIII deficiency. It is expected that recombinant FXIII can be used for the prevention of bleeding episodes.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit)
- Treatment with regular FXIII replacement therapy initiated at least 6 months prior to screening and one of the following : a documented history of at least one 1 treatment-requiring bleeding episode prior to initiation of regular replacement therapy or a documented family history of FXIII congenital deficiency (only for subjects on regular replacement therapy prior to screening)
- Documented history of at least two 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening (only for subjects receiving on-demand treatment prior to screening)
Exclusion Criteria
- Known neutralizing antibodies (inhibitors) towards FXIII
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Documented history of at least 2 treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products (fresh frozen plasma (FFP), plasma-derived FXIII (pd FXIII) and cryoprecipitate)
- Known or suspected allergy to trial product(s) or related products
- Planned major surgery during the trial period. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject
- Renal insufficiency defined as current dialysis therapy
- Any history of confirmed venous or arterial thrombo-embolic events
Data sourced from ClinicalTrials.gov (NCT00713648). 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.