ADCC Mediated B-Cell dEpletion and BAFF-R Blockade

A Randomized, Double-blind, Placebo-controlled Study of the Safety and Efficacy of VAY736 in Autoimmune Hepatitis

Status

Completed

Phases

Phase 2Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03217422

Acronym

AMBER

Enrollment

Registered

2017-07-14

Start date

2018-02-15

Completion date

2025-12-17

Last updated

2026-02-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Autoimmune Hepatitis

Keywords

Type 1, Type 2, autoimmune hepatitis, AIH

Brief summary

VAY736 dose testing; VAY736 efficacy and safety testing.

Detailed description

This is a randomized, placebo-controlled, double-blind dose range study in autoimmune hepatitis. The study population consists of female and male adult autoimmune hepatitis patients with incomplete response or intolerant to standard treatment of care. The diagnosis of autoimmune hepatitis has to fulfill the IAIHG criteria and must be confirmed by liver histology. Patients will be randomly assigned to different doses of VAY736 or placebo. The primary analysis is planned at 24 weeks. A subsequent study part will then test the efficacy and safety of VAY736 in a parallel group design. For this part of the trial a new group of autoimmune hepatitis patients will be enrolled.

Interventions

BIOLOGICALVAY736

VAY736

OTHERPlacebo

Placebo control with conversion to active VAY736

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Masking description

Double blind, placebo-controlled

Eligibility

Sex/Gender

ALL

Age

18 Years to 75 Years

Healthy volunteers

Inclusion criteria

Key Inclusion Criteria: 1. AIH diagnosed per International Autoimmune Hepatitis Group 2. Liver biopsy with Ishak modified HAI indicating active AIH 3. Incomplete response to OR intolerance of standard therapy (per AASLD) Key

Exclusion criteria

1. Prior use of any B-cell depleting therapy (e.g., rituximab or other anti-CD20 mAb, anti-CD22 mAb or anti-CD52 mAb) within 1 year prior to Screening or as long as B-cell count \<50 cells/µL 2. Required regular use of medications with known hepatotoxicity 3. Decompensated cirrhosis 4. Diagnosis of overlap syndrome with AIH (e.g., AIH+PBC, AIH+PSC). 5. Drug related AIH at screening or a history of drug related AIH. 6. History of drug abuse or unhealthy alcohol use 7. History of malignancy of any organ system 8. Pregnant or nursing (lactating) women

Design outcomes

Primary

Measure	Time frame	Description
ALT (Alanine aminotransferase) normalization	Week 24	Difference in ALT normalization

Secondary

Measure	Time frame	Description
ALT normalization by dose	Week 24	VAY736 dose-response

Countries

Argentina, Belgium, Canada, Czechia, Germany, Japan, Spain, Switzerland, United Kingdom, United States

Contacts

STUDY_DIRECTORStudy Director

Novartis Pharmaceuticals

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 28, 2026