Congenital Factor XIII A-subunit Deficiency MedDRA version: 17.0 Level: PT Classification code 10016083 Term: Factor XIII deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Recombinant factor XIII (rFXIII)
Product Code: NN1841
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Catridecacog
CAS Number: 606138-08-3
Current
Sponsors
Novo Nordisk A/S
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Informed Consent obtained before any trial related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the subject.) 2. Previous participation (means up to and including End Of Trial (EOT) visit) in F13CD-3760 Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Known or suspected hypersensitivity to trial product or related products 2. Known history of development of inhibitors against FXIII 3. Hereditary or acquired coagulation disorder other than FXIII congenital deficiency 4. Platelet count (thrombocytes) < 50 x 10 9 / L 5. Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus 6. Previous history of arterial or venous thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis 7. The receipt of any investigational product, except rFXIII, within 30 days of trial enrolment 8. Non-compliant subject judged by the Investigator 9. Any concomitant serious chronic or acute illness or infection expected to impact compliance or safety, judged by the Investigator. 10. Previous participation in this trial. Participation is defined as screened and withdrawn. 11. Medical, social, or psychosocial factors expected to impact compliance or safety 12. Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome including renal and/or liver dysfunction
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long term safety of monthly replacement therapy with rFXIII when used for prevention of bleeding episodes in paediatric subjects with congenital FXIII A-subunit deficiency;Secondary Objective: To investigate the efficacy of monthly replacement therapy with rFXIII when used for prevention of bleeding episodes in paediatric subjects with congenital FXIII A-subunit deficiency;Primary end point(s): Treatment emergent adverse events (serious and non-serious), defined as adverse events occurring from first trial product administration to the end of the subject’s participation in the trial | — |
Countries
Austria, Denmark, Finland, France, Germany, Italy, Spain, Sweden, United Kingdom
Outcome results
None listed