A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to less than 6 Years Old) Subjects with Congenital FXIII A-subunit Deficiency

Phase 1

• Conditions: 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

• Registration Number: EUCTR2009-016869-28-GB
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2010-07-07
• Study Completion: 2012-01-06
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 6

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

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 Participation is defined as dosed and withdrawn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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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);<br> Secondary Objective: • To investigate additional Pharmacokinetic (PK) parameters<br> • 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<br> ;Primary end point(s): Area under the concentration vs. time curve (AUC 0-30 Days) (IU×h/mL)