E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Congenital Factor XIII deficiency
Deficiencia congénita de factor XIII
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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 | 10016083 |
E.1.2 | Term | Factor XIII deficiency |
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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 |
The primary objective of this study is to generate steady-state PK Factor XIII data in subjects with congential Factor XIII deficiency. |
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E.2.2 | Secondary objectives of the trial |
The secondary objective of this study is to assess the safety of Factor XIII administration over a period of 12 weeks in this population. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Severe congential Factor XIII deficiency (< 10 U/dl at diagnosis) 2. Written informed consent 3. Males and females of any age with congenital FXIII deficiency |
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E.4 | Principal exclusion criteria |
1. Diagnosis of acquired Factor XIII deficiency 2. Administration of a Factor XIII- containing product, including blood transfusions or other blood products within 4 weeks prior to the planned day 0 3. Any known congential or acquired coagulation disorder other than congential Factor XIII deficiency 4. Use of any other investigational product within 4 weeks prior to the baseline visit (day 0) 5. Positive result at screening for HIV 6. Negative serology for hepatitis B and has not received a full hepatitis B vaccination 7. Serum aspartate transaminase (AST) or serum alanin transaminase (ALT) concentration > 2.5 times the upper limit of normal 8. Fibrinogen < lower limit of normal 9. Active bleeding 10. Pregnant or breast feeding 11. Intention to become pregnant during the course of the study 12. Female subjects of childbearing potential not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study 13. Surgical procedure anticipated during the study period 14. Suspected inability (e.g. language problems) or unwilingness to comply with study procedures or history of noncompliance |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint in this study is the steady-state PK profile of Factor XIII, measured after the third dose of Factor XIII. |
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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 | Yes |
E.6.3 | Therapy | Yes |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | 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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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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Last visit of the last subject. |
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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 | |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 4 |