071102 - BAX 111 rVWF in Paediatrics
Research type
Research Study
Full title
A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed with Severe von Willebrand Disease. PIP decision numbers P/0091/2012 and P/0214/2015
IRAS ID
218115
Contact name
Judit Koranyi
Contact email
Sponsor organisation
Baxalta Innovations GmbH
Eudract number
2016-001477-33
Clinicaltrials.gov Identifier
N/a, N/a
Duration of Study in the UK
2 years, 8 months, 1 days
Research summary
This is a worldwide phase 3 clinical trial in 27 children (0 - <18yrs) with severe Von Willebrand disease (VWD). Severe VWD is characterised by the lack/malfunctioning blood clotting protein called Von Willebrand Factor (VWF) which causes uncontrolled bleeding. Factor VIII (FVIII) another blood clotting protein can also be affected in VWD. There are two drugs in the trial BAX111 a recombinant (i.e. made in a lab) VWF and ADVATE which is FVIII replacement. BAX111 is licensed for on demand treatment for adults with VWD in the US. ADVATE is licensed for treatment of haemophilia in US, Canada and Europe but not VWD. Both drugs are investigational drugs in this trial.
There are 3 treatment arms in the trial - On Demand, Planned Surgery and Emergency Surgery. Participants either receive BAX111 or BAX111 and ADVATE. It is an open label study meaning participants parents and their doctor know what treatment your child will receive and Phase 3 means the drug has been tested in children before but is being tested in a larger number of children. The trial looks at how well the bleeding is controlled in patients not having surgery (on demand group) and having surgery (planned and emergency surgery groups).
The study drug are given via a slow injection into a vein (infusion), treatment will be given at hospital to begin but may be able to be given at home later on. Participants would be in the trial for approximately 14 months, the number of visits to hospital depend of the treatment arm. Participants on the surgery arms will rollover onto the on demand treatment after surgery if spaces are available.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
16/WM/0511
Date of REC Opinion
22 Feb 2017
REC opinion
Further Information Favourable Opinion