E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Congenital FXIII A-subunit Deficiency |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 13.1 |
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 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) |
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E.2.2 | Secondary objectives of the trial |
• 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
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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
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E.4 | Principal 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 Participation is defined as dosed and withdrawn 4.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
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E.5 End points |
E.5.1 | Primary end point(s) |
Area under the concentration vs. time curve (AUC 0-30 Days) (IU×h/mL) |
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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 | No |
E.6.6 | Pharmacokinetic | Yes |
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 |
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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 |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |