A Multi-Centre, Multinational, Open-Label, Single-Arm and Multiple Dosing Trial on Safety and Efficacy of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Paediatric Subjects with Congenital Factor XIII A-subunit Deficiency Safety Extension Trial to F13CD-3760
- Conditions
- Congenital Factor XIII A-subunit DeficiencyMedDRA version: 17.0Level: PTClassification code 10016083Term: Factor XIII deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
- Registration Number
- EUCTR2010-020192-23-GB
- Lead Sponsor
- ovo Nordisk A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 6
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
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 Participation is defined as screened and withdrawn.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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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
- Secondary Outcome Measures
Name Time Method