E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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 | 10047715 |
E.1.2 | Term | Von Willebrand's disease |
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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 efficacy of OPTIVATE® in preventing excessive blood loss during and after surgery. |
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E.2.2 | Secondary objectives of the trial |
To assess the safety of OPTIVATE® during and after surgery. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Have given written informed consent. 2. Be aged 12 years or older. 3. Have VWD of known type. 4. Be due to undergo surgery, in which the investigator believes a VWF concentrate will be required. 5. Have a known lack of, or poor response to DDAVP, or require a specific type of surgery in which a plasma-derived product is appropriate. 6. Have a prothrombin time (PT) of not more than 3 seconds above the upper limit of the reference range. 7. Female patients of child-bearing potential, with the exception of pregnant patients undergoing Caesarean surgery or normal delivery, must have a negative result on a human chorionic gonadotropin-based pregnancy test. If a female patient is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study. |
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E.4 | Principal exclusion criteria |
1. Have a history of inhibitor development to VWF or FVIII or a positive result at screening. A result at screening is not mandatory if the patient is to undergo emergency surgery and the local laboratory is unable to perform the analyses. 2. Patients with thrombocytopenia (platelets <50 x 10 to the power of 9/L). 3. Patients who have clinically significant renal disease creatinine >200 μmol/L). 4. Patients who have clinically significant liver disease (ALT levels greater than three times the upper limit of the reference range). 5. Presence of any other major systemic illnesses which would compromise the outcome of the study in the opinion of the investigator. 6. Known or suspected hypersensitivity to investigational medicinal product (IMP) or its excipients. 7. Have a recent history of alcohol or drug abuse. 8. Administration of a new chemical entity within the 4 months preceding enrolment. 9. Participation in any other clinical study in which investigational or marketed drugs were employed in the 30 days preceding enrolment (screening visit) into this study, with the exception of the BPL clinical study Protocol 8VWF01 or earlier participation in this study for a separate surgical procedure. 10. Female patients who are lactating. 11. In the opinion of the investigator, the patient is unlikely to comply with the study protocol. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary end point is to assess the efficacy of OPTIVATE in preventing excessive blood loss, which will be will be assessed by a subjective assessment by the investigator of Optivate in the control of bleeding due to surgery throughout the whole study. |
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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 | 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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 8 |
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 | 8 |
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 |