rVWF:rFVIII in Type 3 Von Willbrand Disease

• Research type

  Research Study

• Full title

  RECOMBINANT VON WILLEBRAND FACTOR / RECOMBINANT FACTOR VIII COMPLEX (rVWF:rFVIII): A PHASE 1 STUDY EVALUATING THE PHARMACOKINETICS, SAFETY AND TOLERABILITY IN TYPE 3 VON WILLEBRAND DISEASE

• IRAS ID

  11379

• Sponsor organisation

  Baxter Innovations GmbH

• Eudract number

  2008-000047-34

• ISRCTN Number

  0

• Clinicaltrials.gov Identifier

  0

• Research summary

  This phase I clinical trial is for patients with type 3 Von Willebrand Disease (VWD) a disease in which the body is unable to make the blood protein called Von Willbrand Factor (VWF), which is required to stop bleeding when you injure yourself. Approximately 1% of VWD patients exhibit a total or near total absence of VWF, which is classified as type 3 VWD. Baxter Healthcare Corporation has developed a human recombinant von Willebrand Factor (rVWF). Recombinant means that the products will be produced by a cell line outside of the human body. The disease will be treated with VWF concentrates derived from human plasma, which usually contains factor VIII aswell. Factor VIII is a protein circulating in the blood, which is required to stop bleeding when you injure yourself. In addition it also protects the VWF. Therefore all commercial available VWF concentrates contains factor VIII aswell.The main purpose of the study is to find out the immediate tolerability and safety in humans of single doses of rVWF:rFVIII after it has been administered at different dose levels of 2, 7.5, 20 and 50 IU/Kg VWF and how the body reacts and breaks down the study drug with plasma derived product after its infusion. The other aim is to compare the distribution in the body of the recombinant study drug with plasma derived product after infusion in the last part of the study (Cohort 4). A total of 31 subjects will be enrolled in centres in Europe and the US. As there is no human experience with rVWF:rFVIII, a dose escalation study design consisting of 4 cohorts will be employed. Cohort 1 comprises three patients, who will receive the lowest dose. Cohort 1 will be taking place in USA and Austria only. Cohort 2 comprises five patients with a higher dose of VWF. Cohort 3 comprises further five patients, who will be included in Europe and the US. Cohort 4 compromises approximately 18 patients who will receive the highest dose of VWF. The benefit for this study may include a better understanding of the pharmacology of VWF and related FVIII pharmacokinetics and also the availability of a recombinant product with no risk of contamination of unknown viruses.

• REC name

  North West - Haydock Research Ethics Committee

• REC reference

  08/H1010/109

• Date of REC Opinion

  4 Mar 2009

• REC opinion

  Further Information Favourable Opinion