RVWF TREATMENT OF BLEEDS

• Research type

  Research Study

• Full title

  A A Phase 3 clinical study to determine the safety and efficacy of rVWF:rFVIII and rVWF in the treatment and prevention of bleeding episodes in subjects diagnosed with von Willebrand disease

• IRAS ID

  82159

• Contact name

  Thynn Thynn Yee

• Sponsor organisation

  Baxter Innovations GmbH, Industriestr. 67, 1220 Vienna, Austria

• Eudract number

  2010-024108-84

• ISRCTN Number

  no issue

• Research summary

  People with Von Willebrand Disease (VWD) are unable to make the blood protein Von Willebrand Factor (VWF). VWF stops bleeding together with Factor VIII (FVIII).Baxter has developed a new medicine called recombinant human VWF (rVWF). VWD patients are currently treated with plasma derived (pd)VWF and rFVIII. Plasma derived means the medicine is made from human or animal sources; recombinant means it??s made in a laboratory. It is hoped that rVWF will be safer than pdVWF because the risk of contamination with viruses or Creutzfeldt-Jakob Disease is theoretically eliminated. The purpose of the study is to find out the following about rVWF when given alone or with rFVIII: 1. How the body reacts and breaks down rVWF (PK assessments). 2. How safe and effective rVWF and rFVIII is for the prevention and treatment of bleeding episodes at different doses, and how well these doses are tolerated immediately after dosing. 3. Any changes in health-related quality of life. Approximately 40 people with severe VWD will participate from sites worldwide. The study consists of 2 parts: A and B. Part A has 2 PK assessments and a treatment period of 6 months. Part B consists of continued treatment (for bleeding events) for an additional 6 months. Participants will be in the study for approximately 14 months.The rVWF and rFVIII will be given directly into a vein (by intravenous infusion). All participants will be monitored closely for up to 4 days following the PK assessments; there will also be a follow-up visit a few weeks after each PK assessment. In addition, participants will need to visit the study site every 3 months and for the treatment of bleeding episodes. Study visits will include blood tests, vital signs, ECGs and questionnaires.

• REC name

  London - Bloomsbury Research Ethics Committee

• REC reference

  11/LO/1312

• Date of REC Opinion

  31 Oct 2011

• REC opinion

  Further Information Favourable Opinion