E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10052474 |
E.1.2 | Term | Factor X deficiency |
|
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 pharmacokinetics of Factor X after a single dose of 25 IU/kg |
|
E.2.2 | Secondary objectives of the trial |
To assess the efficacy of Factor X in the treatment of bleeding episodes over at least 6 months. To assess the safety of Factor X in the treatment of bleeding episodes over at least 6 months. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Informed consent At least 12 years of age With hereditary severe or moderate factor X deficiency (<5% basal activity at diagnosis) Currently treated with fresh frozen plasma (FFP), prothrombin complex concentrates (PCC) or factor IX/X concentrate Have had a minimum of one spontaneous or menorrhagic bleed in the last 12 months which required treatment with either FFP, PCC or factor IX/X concentrate At least 14 days since an infusion of FFP, PCC or factor IX/X concentrate at the Baseline Visit Female subjects of childbearing potential must not be pregnant at entry to the study and must practice contraception for the duration of the study. |
|
E.4 | Principal exclusion criteria |
History of inhibitor development to FX, or a positive inhibitor result at the Screening Visit Bleeding at the Baseline Visit Subjects with thrombocytopenia, clinically significant renal disease or clinically significant liver disease Subjects with other coagulopathy or thrombophilia Known or suspected hypersensitivity to the investigational medicinal product or its excipients Known to have abused chemicals or drugs within the past 12 months History of unreliability or non-cooperation Participation in another clinical trial within the past 30 days, with the exception of BPL FX surgery study (protocol No Ten03). In such cases, subjects should have completed their End-of-Study Visit either before or on the day of the Screening Visit for this study Female subjects who are pregnant or lactating. Subjects who are planning more than 4 weeks absence from the locality of the investigational site, between the Screening Visit and the Repeat PK Assessment. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The PK parameters incremental recovery, half-life (non-compartmental), half-life (two compartmental, if appropriate), AUC (0-144h), AUC (0-infinity), clearance, MRT (0-infinity) volume of distribution, Cmax, Cmax(obs), Tmax and terminal elimination rate constant for FX:C at Baseline Visit and 6 Month Visit. |
|
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 | 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 | Yes |
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 | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
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 |
|
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
|
See protocol Section A-4.4. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
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 | 2 |
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 |