AMBER: VAY736 in Autoimmune Hepatitis
Research type
Research Study
Full title
A two-part randomized, double-blind, placebo-controlled multicenter dose ranging and confirmatory study to assess the safety and efficacy of VAY736 in autoimmune hepatitis patients with incomplete response to or intolerance of standard therapy(AMBER)
IRAS ID
236707
Contact name
Dania Calboli
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2017-001555-32
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 11 months, 17 days
Research summary
Autoimmune hepatitis (AIH) is a rare, long-term autoimmune disease, the causes of which are unknown. In AIH the immune system attacks and damages the liver causing inflammation (hepatitis). Although a rare condition, AIH can lead to poor health and lethal outcomes resulting from liver failure. Approximately 25% of AIH patients do not respond to conventional treatments which target suppression of liver inflammation. Better tolerated treatments are needed to control disease activity in AIH patients.
VAY736 belongs to a class of drugs called monoclonal antibodies which are protein drugs that are able to recognize, target and stick to specific proteins on specific cells. VAY736 binds to a protein on B-cells, a group of white blood cells in the immune system, which play an important role in AIH. Approximately 101 patients have received VAY736 in clinical studies to date, but no AIH patients have yet received VAY736.
The study design is a two-part, randomized, double-blind, placebo-controlled multi-centre trial to understand:
•If VAY736 can help to treat patients with AIH who aren’t responding to or cannot tolerate standard therapy
•If it is safe for AIH patients to take VAY736
•Which dose of VAY736 works best
•If using VAY736 in addition to standard treatment is better than standard treatment alone.Patients will participate in the study for a minimum of about 76 weeks (approximately 18 months, including the 4-week screening period, the 52 week treatment period and the minimum 20 week follow-up period). The maximum time patients will be participating is 3 years if additional monitoring of the patient’s B-cells is required. 80 AIH patients will be recruited worldwide for part 1 of the study and if this part of the study is successful another 280 patients will be recruited for part 2 of the study.
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
17/WM/0445
Date of REC Opinion
16 Jan 2018
REC opinion
Further Information Favourable Opinion