Congenital FXIII A-subunit Deficiency MedDRA version: 13.1 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: NN 1841
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed
Sponsors
Novo Nordisk A/S
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Signed Informed Consent by subject’s parents or subject’s legally acceptable representative before any trial related activities. Trial related activities are any procedures that would not have been performed during the normal management of the subject 2. Age 1 to less than 6 years old at the time of enrolment 3. Congenital FXIII subunit-A deficiency previously documented by genotyping or evaluated by genotyping through blood sampling at screening visit 4. Body weight = 10 kg 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 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Received blood products or FXIII concentrates within 4 weeks of trial product administration 2. Known antibodies to FXIII 3. Previous participation in this trial. Participation is defined as dosed and withdrawn 4. Hereditary or acquired coagulation disorder other than FXIII A-subunit congenital deficiency 5. Platelet count (thrombocytes) < 50 × 10 9/L (at screening visit) 6. Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus 7. Previous history of arterial or venous thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis 8. Administration of any antithrombotic or antiplatelet drugs within 7 days of trial enrolment 9. Known or suspected allergy to trial product or related products 10. Received treatment with any experimental agent within 30 days of trial enrolment 11. Any concurrent serious chronic or acute illness or infection expected to impact compliance or safety as judged by the investigator 12. Any surgical procedure in the 30 days prior to enrolment and any planned surgery during the trial period 13. Medical, social, or psychosocial factors expected to impact compliance or safety 14. Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject or interfere with the trial participation or trial outcome including renal and/or liver dysfunction
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To characterise the pharmacokinetics of rFXIII in paediatric subjects (1 to less than 6 years old) with congenital FXIII A-subunit deficiency following a single intravenous dose administration by measuring the area under the concentration vs. time curve (AUC0-30 Days) (IU×h/mL); Secondary Objective: • To investigate additional Pharmacokinetic (PK) parameters • To evaluate the safety of a single intravenous dose of rFXIII in paediatric subjects (1 to less than 6 years old) with congenital FXIII A-subunit deficiency ;Primary end point(s): Area under the concentration vs. time curve (AUC 0-30 Days) (IU×h/mL) | — |
Countries
United Kingdom
Outcome results
None listed