E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Congenital Factor XIII Deficiency |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10010432 |
E.1.2 | Term | Congenital deficiency of other clotting factors |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10061992 |
E.1.2 | Term | Haemophilia |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10009735 |
E.1.2 | Term | Coagulation disorders congenital |
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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 efficacy of monthly replacement therapy with rFXIII on prevention of bleeding episodes in subjects with congenital FXIII deficiency. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the safety of monthly replacement therapy with rFXIII. |
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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. Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit). 3. Treatment with regular FXIII replacement therapy initiated at least 6 months prior to screening and one of the following : a documented history of ≥1 treatment-requiring bleeding episode prior to initiation of regular replacement therapy or a documented family history of FXIII congenital deficiency. 4. Documented history of ≥ 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening. 5. Subjects with age ≥ 6 years and a weight ≥20 kg. Before enrolling subjects ≥ 6 to < 12 years of age in the EU countries, 7 subjects have to be exposed for 12 weeks (3 exposures) to trial product with a safe safety profile. 6. If female and of child-bearing potential: negative pregnancy test at screening.
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E.4 | Principal exclusion criteria |
1. Known neutralizing antibodies (inhibitors) towards FXIII. 2. Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency. 3. Documented history of ≥ 2 treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products (FFP, pd FXIII and cryoprecipitate). 4. Platelet count (thrombocytes) < 75 × 10^9/L. 5. Known or suspected allergy to trial product(s) or related products. 6. Previous participation in this trial. 7. Subject has received treatment with any investigational drug within 30 days of trial enrolment, except pdFXIII. 8. Planned major surgery during the trial period. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject. 9. Renal insufficiency defined as current dialysis therapy. 10. Any history of confirmed venous or arterial thrombo-embolic events. 11. Subject has received any anti-thrombotic or anti-platelet drugs within 7 days of trial enrollment. 12. Subject has medical, social or psychosocial factors expected to impact compliance or safety. 13. 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. 14. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation in participating in the trial. 15. Females of childbearing potential who are pregnant, breastfeeding or intend to become pregnant or are not using adequate contraceptive methods (adequate contraceptive measures as required by local law or practice) from the time of enrollment to completion of all follow-up trial visits, if there is any risk of pregnancy in the opinion of the investigator.
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E.5 End points |
E.5.1 | Primary end point(s) |
•Rate (number per subject year) of ”bleeding episodes requiring treatment” with a FXIII containing product during the rFXIII treatment period |
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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 | Yes |
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 | Yes |
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 | No |
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 | Yes |
E.8.2.3.1 | Comparator description |
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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 | 10 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
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 | 9 |
E.8.9.1 | In the Member State concerned days | |
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 | 9 |