BAX855 PUP study
Research type
Research Study
Full title
Phase 3, prospective, multi-center, open label study to investigate safety, immunogenicity, and hemostatic efficacy of PEGylated Factor VIII (BAX 855) in previously untreated patients (PUPs) < 6 years with severe hemophilia A (FVIII < 1%)
IRAS ID
189003
Contact name
John Grainger
Contact email
Sponsor organisation
Baxalta Innovations GmbH
Eudract number
2015-002136-40
Duration of Study in the UK
8 years, 0 months, 0 days
Research summary
Summary of Research
This is a Phase 3 study of study drug BAX855. BAX855 has already been tested in humans but is being tested in a larger number of patients; to investigate the safety of study drug BAX 855, how well it works in treating and preventing bleeds, and whether it provokes an immune response (reaction of the body to a foreign substance that is not usually found in the body) in children with severe haemophilia A who previously either have not received any treatment or have received minimal treatment.
BAX855 is a laboratory made blood-clotting factor similar to ADVATE, an approved medication in many countries. BAX855 is a form of ADVATE that has been modified, by the attachment of polyethylene glycol (PEG). The aim of adding the PEG is to extend the time the medication stays in the bloodstream to allow for less frequent dosing in the treatment of haemophilia A.
The study drug is intended for use as “prophylaxis” (a fixed infusion [slow injection into a vein] regimen to prevent bleeding), or for “on-demand” use (infusion to treat or prevent bleeding in case of surgery).
In some patients, BAX855 can provoke an immune response or immune reaction, and “inhibitors” of the study drug may develop. Inhibitors are also known as “neutralising antibodies” because they cancel out the effect of their target in this case BAX855. Only patients who develop these study drug inhibitors will be asked to have a different dose of the study drug to see if this helps. The Main part of study, participation will be approximately 2 years, depending on the type of treatment. If the patient proceeds to have treatment for antibodies to BAX855, the study will last for up to 5 years in total.Summary of Results
The lay summary of study results will be published on Takedas Clinical Trial website (not yet published)URL: https://eur03.safelinks.protection.outlook.com/?url=https%3A%2F%2Ftrack.pstmrk.it%2F3ts%2Fclinicaltrials.takeda.com%252Fstudy-detail%252F5f6b5fc74db2bf003ab45e99%2FNBTI%2FG3G9AQ%2FAQ%2F5ae7214a-a309-48b0-9388-f2efecd59527%2F2%2F1LKggF0lUr&data=05%7C02%7Capprovals%40hra.nhs.uk%7Ce41f4f88b1c44315a1b008dd96d718a3%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638832572776894004%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=T%2FiqvRiqcC0%2FeSi0XaeaJKqC4RF8X8ks8UgfsfCgY94%3D&reserved=0
REC name
West of Scotland REC 1
REC reference
15/WS/0216
Date of REC Opinion
26 Oct 2015
REC opinion
Further Information Favourable Opinion