E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Congenital Factor XIII A-subunit Deficiency |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10016083 |
E.1.2 | Term | Factor XIII deficiency |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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 |
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E.2.2 | Secondary objectives of the trial |
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 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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
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E.4 | Principal 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
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E.5 End points |
E.5.1 | 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 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Single arm and multiple dosing |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 5 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Denmark |
France |
Italy |
Austria |
Sweden |
Finland |
Germany |
Spain |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |