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 |
Pharmacokinetics of Optivate |
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E.2.2 | Secondary objectives of the trial |
Efficacy and Safety of Optivate in long-term use over at least 12 months. |
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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 severe VWD (VWF:RCo <20%) of known type. Severity will be confirmed by a current VWF:RCo result of <20%. 4. Be known or expected to require a concentrate for management of VWD. 5. Must have had at least one spontaneous bleed in the last 12 months which required treatment with a FVIII and VWF concentrate. 6. Have a known lack of, or poor response to DDAVP, or for whom DDAVP is contraindicated. 7. Have a prothombin time (PT) of not more than 3 seconds above the upper limit of the reference range. 8. At the Baseline Visit (Visit 1), patients must have had at least 5 days since their last infusion of replacement factor concentrate or DDAVP (see section 9.3.10 regarding the use of other concomitant medication). 9. Female patients of child-bearing potential must have a negatice 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. Female patients must not be lactating. |
|
E.4 | Principal exclusion criteria |
1. Have a history of inhibitor development to VWF or FVIII or a positive result at screening. 2. Actively bleeding (Note: the patient can enter the study once the bleed is controlled). 3. Presence of major systemic illnesses: renal disease, liver disease, or neurological or psychiatric disease which would compromise the outcome of the study in the opinion of the investigator. 4. Known or suspected hypersensitivity to investigational medicinal product (IMP) or its excipients. 5. Have a recent history of alcohol or drug abuse. 6. Administration of a new chemical entity within the 4 months preceding enrolment. 7. Participation in any other clinical study in which investigational or marketed drugs were employed in the 30 days preceding enrolment into this study, with the exception of the BPL clinical study protocol 8VWF03, in such cases patients should have completed their End-of study visit either before or on the day of screening for this study. 8. In the option of the investigator, the pateint is unlikely to comply with the study protocol. |
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E.5 End points |
E.5.1 | Primary end point(s) |
AUC (0-t), AUC(0-72h), MRT(0-t) and MRT (0-72h) for VWF:RCo at the Baseline Visit (Visit 1) by VWD type and overall |
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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 | 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 | 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.8.3 |
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 | 7 |
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
|
12 month treatment of last patient |
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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 | 18 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 18 |