E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Factor XIII Congenital 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 | HLGT |
E.1.2 | Classification code | 10064477 |
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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 | 10009737 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10016083 |
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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 assess the long term safety of monthly replacement therapy with rFXIII when used for 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 efficacy of monthly replacement therapy with rFXIII when used for prevention of bleeding episodes in subjects with congenital FXIII 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 inclusive Visit 16, (EOT)) in F13CD-1725. 3. If female and of childbearing 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 ≥ 3 spontaneous and haemostatic treatment-requiring bleeding episodes per year during previous regular replacement therapy with rFXIII 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. (Defined as screened at visit 1). 7. Treatment with any investigational drug within 30 days of trial enrolment, except pdFXIII and rFXIII 8. Renal insufficiency defined as currently requiring dialysis therapy. 9. Any history of confirmed venous or arterial thrombo-embolic events, including myocardial infarction or stroke 10. Medical, social or psychosocial factors expected to impact compliance or safety. 11. 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. 12. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation in participating in the trial. 13. Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods (adequate contraceptive measures as required by local law or practice) from the time of enrolment 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) |
Adverse events (serious and non serious) occurring bform first trial related activity after signing the informed consent to the end of 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 | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 23 |
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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I soggetti parteciperanno allo studio fino a che il prodotto sperimentale non ricevera`l`autorizzazione alla messa in commercio.In ogni caso, periodo minimo di partecipazione e trattamento di un soggetto sara` di 52 settimane. |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 8 |