Congenital Factor XIII Deficiency MedDRA version: 9.1 Level: LLT Classification code 10010432 Term: Congenital deficiency of other clotting factors MedDRA version: 9.1 Level: LLT Classification code 10061992 Term: Haemophilia MedDRA version: 9.1 Level: HLT Classification code 10009735 Term: Coagulation
Conditions
Interventions
Product Name: Recombinant Factor XIII (rFXIII)
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: Catridecacog
CAS Number: 6061
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. Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit). 3. Treatment with regular FXIII replacement therapy initiated at least 6 months prior to screening and one of the following : a documented history of =1 treatment-requiring bleeding episode prior to initiation of regular replacement therapy or a documented family history of FXIII congenital deficiency. 4. Documented history of = 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening. 5. Subjects with age = 6 years and a weight =20 kg. Before enrolling subjects = 6 to =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Known neutralizing antibodies (inhibitors) towards FXIII. 2. Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency. 3. Documented history of = 2 treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products (FFP, pd FXIII and cryoprecipitate). 4. Platelet count (thrombocytes) < 75 × 10^9/L. 5. Known or suspected allergy to trial product(s) or related products. 6. Previous participation in this trial. 7. Subject has received treatment with any investigational drug within 30 days of trial enrolment, except pdFXIII. 8. Planned major surgery during the trial period. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject. 9. Renal insufficiency defined as current dialysis therapy. 10. Any history of confirmed venous or arterial thrombo-embolic events. 11. Subject has received any anti-thrombotic or anti-platelet drugs within 7 days of trial enrollment. 12. Subject has medical, social or psychosocial factors expected to impact compliance or safety. 13. 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. 14. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation in participating in the trial. 15. Females of childbearing potential who are pregnant, breastfeeding or intend to become pregnant or are not using adequate contraceptive methods (adequate contraceptive measures as required by local law or practice) from the time of enrollment to completion of all follow-up trial visits, if there is any risk of pregnancy in the opinion of the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of monthly replacement therapy with rFXIII on prevention of bleeding episodes in subjects with congenital FXIII deficiency.;Secondary Objective: To evaluate the safety of monthly replacement therapy with rFXIII.;Primary end point(s): •Rate (number per subject year) of ”bleeding episodes requiring treatment” with a FXIII containing product during the rFXIII treatment period | — |
Countries
Austria, Finland, France, Germany, Italy, Spain, United Kingdom
Outcome results
None listed