E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Inherited von Willebrand Disease |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 8.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10047715 |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this clinical study is to assess the efficacy of WILATE for the prevention and/or treatment of bleedings, and in surgical procedures. |
|
E.2.2 | Secondary objectives of the trial |
Secondary objectives are the determination of inhibitors to VWF and FVIII and to assess the clinical safety and tolerability of treatment with WILATE. Recovery assessment will be performed in patients undergoing a major surgery. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Defined inherited VWD of any type. Age <6 years at study admission. DDAVP treatment known or suspected to be inadequate, insufficient or contraindicated. An expected minimum of 5 exposure days to WILATE within 1 year of observation. HIV-1/2 negative. Freely given fully informed consent has been obtained from the patient's parents (in accordance with local laws, the informed consent of both parents might be necessary). |
|
E.4 | Principal exclusion criteria |
Any haematological disorder other than VWD. Diagnosis of acquired VWD. Any known present or past inhibitor activity against VWF or FVIII. Administration of DDAVP or other blood/plasma products 5 days prior to the 1st WILATE injection Administration of acetylsalicylic acid 14 days before treatment with Wilate. Known history of intolerance towards plasma derived or blood products. Participation in another clinical study currently or during the past four weeks. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy of WILATE in prevention (prophylactic treatment) and/or treatment of bleeding episodes measured by: the number of bleedings, amount of IMP required (consumption of WILATE), number of exposures necessary to stop bleedings, assessment of response to treatment of bleeding episodes. Overall efficacy will be recorded using a 4-point verbal rating scale (VRS). The details of all treatments and bleedings will be documented. Efficacy of WILATE in surgical procedures measured by: amount of IMP used , haemostasis reached, loss of blood (intra- and postoperatively), the requirements of additional blood or plasma transfusions. Overall efficacy will be recorded using a 4-point verbal rating scale (VRS). Details of all treatments and characteristics of the surgical procedures will be documented. |
|
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 | 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 | Information not present in EudraCT |
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 | 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 | 6 |
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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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
|
End of the study is the last patient's last visit |
|
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 | 6 |
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 | 6 |