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

ISRCTN	ISRCTN12436735
DOI	https://doi.org/10.1186/ISRCTN12436735
EudraCT/CTIS number	2008-001910-25
Secondary identifying numbers	WIL-21

Submission date: 01/09/2008
Registration date: 04/09/2008
Last edited: 20/05/2019

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Haematological Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English Summary

Not provided at time of registration

Contact information

Ms Martina Jansen
Scientific

Oberlaaerstrasse 235
Vienna
1100
Austria

Phone	+43 (0)1 61032 1208
Email	martina.jansen@octapharma.com

Study information

Study design	Prospective, randomised, controlled, open-labelled, two-arm cross-over, multi-centre phase II study
Primary study design	Interventional
Secondary study design	Randomised controlled trial
Study setting(s)	Hospital
Study type	Treatment
Participant information sheet	Not available in web format, please use the contact details below to request patient information material
Scientific title	Pharmacokinetics of Wilate® and Haemate® P in von Willebrand type 3 patients: a prospective, randomised, controlled, open-labelled, two-arm cross-over study
Study hypothesis	Comparison of pharmacokinetics of Wilate® and Haemate® P in von Willebrand type 3 patients.
Ethics approval(s)	Ethics approval received from: 1. Ethics Committee of SHAT "Joan Pavel" OOD hospital, Sofia on the 15th July 2008 2. Ethics Committee of FNsP Cyril and Method Hospital, Bratislava on the 24th June 2008
Condition	von Willebrand disease
Intervention	1. Wilate® vials of approximately 400 IU VWF:RCo 2. Haemate® P vials of approximately 500 VWF:RCo One vial of freeze-dried concentrate is reconstituted in 0.1% polysorbate 80 solution/water for injection. A dose of at least 40 IU VWF:RCo/kg body weight will be given intravenously by bolus administration. Each product is administered once. Blood samples will be taken at the following time-points after each infusion: 30 minutes before and at 15 minutes, 30 minutes, 45 minutes, 1, 3, 6, 12, 24, 30, 48, and 72 hours after the infusion.
Intervention type	Drug
Pharmaceutical study type(s)
Phase	Phase II
Drug / device / biological / vaccine name(s)	Wilate®, Haemate® P
Primary outcome measure	The in-vivo half life (t1/2) of Wilate® is the primary endpoint and will be calculated for VWF ristocetin cofactor activity (VWF:RCo), FVIII:C, VWF antigen (VWF:Ag), and VWf collagen binding assay (VWF:CB). Outcomes will be determined from plasma levels measured from samples taken at the above mentioned time-points.
Secondary outcome measures	1. Pharmacokinetics: the following parameters are secondary endpoints and will be calculated for VWF:RCo, FVIII:C, VWF:Ag and VWF:CB: 1.1. Area under the curve (AUC) 1.2. Maximum plasma concentration (Cmax) 1.3. Time to reach maximum plasma concentration (Tmax) 1.4. Mean residence time (MRT) 1.5. Volume of distribution (Vd) 1.6. Clearance (Cl) For the calculation of these parameters, the labelled potency of the respective drug will be the taken. Outcomes will be determined from plasma levels measured from samples taken at the above mentioned time-points. 2. Recovery: in vivo incremental recovery of VWF:RCo, FVIII:C, VWF:Ag and VWF:CB will be calculated from the levels before and after the injection. For the calculation of recovery, the labelled potency of the respective drug will be the taken. 3. Tolerability: tolerability will be assessed by monitoring vital signs, haematological parameters (red blood cell [RBC] count, white blood cell [WBC] count, haemoglobin, haematocrit [HCT], and platelet count [PC]), and by monitoring adverse events (AEs)
Overall study start date	01/09/2008
Overall study end date	01/11/2008

Eligibility

Participant type(s)	Patient
Age group	Not Specified
Sex	Both
Target number of participants	6
Total final enrolment	9
Participant inclusion criteria	1. Defined inherited von Willebrand disease (VWD) type 3 2. 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. Negative for hepatitis B surface antigen (HBsAg) 4. For human immunodeficiency virus (HIV)-positive subjects: must have a baseline CD4+ cell count of greater than 200/mm^3, and a platelet count of greater than 100,000/dL 5. Freely given 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
Participant exclusion criteria	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 von Willebrand factor (VWF)/coagulation factor eight (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
Recruitment start date	01/09/2008
Recruitment end date	01/11/2008

Locations

Countries of recruitment

Austria
Bulgaria
Slovakia

Study participating centre

Oberlaaerstrasse 235

Vienna
1100
Austria

Sponsor information

Octapharma AG (Switzerland)
Industry

Seidenstrasse 2
Lachen
8853
Switzerland

Phone	+41 (0)55 4512121
Email	olaf.walter@octapharma.ch
Website	http://www.octapharma.com
"ROR"	https://ror.org/002k5fe57

Funders

Funder type

Industry

Octapharma AG (Switzerland)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Basic results			20/05/2019	No	No

Editorial Notes

The following changes were made to the trial record:
1. Added clinicaltrialsregister.eu link to basic results (scientific).
2. The total final enrollment was added.