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Pharmacokinetics of WILATE® and Haemate® P in von Willebrand type 3 patients - a prospective, randomised, controlled, open-labelled, 2-arm cross-over study

Phase 1
Conditions
Inherited von Willebrand Disease (VWD) type 3
MedDRA version: 9.1Level: LLTClassification code 10047715Term: Von Willebrand's disease
Registration Number
EUCTR2008-001910-25-SK
Lead Sponsor
Octapharma AG
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
6
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.

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: 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.;Secondary Objective: 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.;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.<br>The calculations will be made by applying non-compartmental pharmacokinetic methods determined from plasma levels obtained at pre-defined time points.<br>
Secondary Outcome Measures
NameTimeMethod
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