E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Inherited von Willebrand Disease (VWD) type 3 |
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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 | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine the half-life of WILATE® in terms of the ristocetin cofactor activity (VWF:RCo), the FVIII coagulant activity (FVIII:C), the VWF antigen (VWF:Ag), and collagen binding activity (VWF:CB) of WILATE® and to compare these parameters with those for Haemate® P. |
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E.2.2 | Secondary objectives of the trial |
To compare the pharmacokinetic profile (PK) of WILATE® with that of Haemate® P; To calculate the incremental recovery of VWF:RCo, FVIII:C, VWF:Ag, and VWF:CB; To assess the tolerability. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
To qualify for enrolment, each subject must satisfy the following criteria before study entry: 1. Must have defined inherited VWD type 3. 2. Must be a male or female subject of at least 12 years of age and have a body weight of at least 32 kg but not more than 125 kg. 3. Be negative for HBsAg. 4. For HIV-positive subjects: must have a baseline CD4+ cell count of >200/mm3, and a platelet count of >100,000/dL. 5. Freely give written informed consent. For subjects who are not legally permitted to provide written consent, the consent must be provided by parents or legal guardians. 6. Females must promise to avoid becoming pregnant for Visits 1 to 11.
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E.4 | Principal exclusion criteria |
Subjects will not be included if any of the following exclusion criteria are met: 1. Subjects with any other bleeding disorders. 2. Known history of intolerance to plasma derivatives or blood products. 3. Present or past inhibitor activity directed against any VWF/FVIII component. 4. Severe liver or kidney disease. 5. Participation in another clinical study involving an investigational treatment, either currently or within the 4 weeks prior to study entry. Studies consisting of data and blood sampling collections on a regular or long-term basis are exempt from this exclusion. 6. Subjects with excessive alcohol or illicit drug usage. 7. Subjects who cannot comply with protocol requirements. 8. Pregnant or lactating women.
Before administration of one of the studied drugs, the following exclusion criteria must be checked and if any of these criteria are met the subject may not be treated at this point of time: 1. Subject in active bleeding state. 2. Administration of plasma, other plasma derivatives, blood products, cryoprecipitate, or DDAVP within 7 days before study drug injection. 3. Administration of acetylsalicylic acid or other NSAIDs within 7 days prior to study drug injection.
4. Before the 2nd study drug administration only (i.e. Visit 7): 4a) Previous study drug injection took place less than 7 days or 4b) more than 4 weeks ago. If any of the criteria 4. – 4a) are met, the PK assessment has to be postponed, however, the subject may stay in the study. Subjects who meet criteria 4b) will be withdrawn from the study
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E.5 End points |
E.5.1 | Primary end point(s) |
The in-vivo half life (t½) of WILATE® is the primary endpoint and will be calculated for VWF:RCo, FVIII:C, VWF:Ag, and VWF:CB. The calculations will be made by applying non-compartmental pharmacokinetic methods determined from plasma levels obtained at pre-defined time points.
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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 | No |
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 | Yes |
E.8.1.1 | Randomised | Yes |
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 | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
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
| 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 | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
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
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The study recruitment phase (first subject in - last subject in) is estimated to be 3 months starting Q2 2008. The study duration per subject will be between 2.5 and 8 weeks. Visit #11 ends the Study. Visit #11 is the last visit for this Study. |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 5 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 5 |
E.8.9.2 | In all countries concerned by the trial days | 0 |