Autoimmune HepatitisPhase 2/3AMBERNCT03217422
ADCC Mediated B-Cell dEpletion and BAFF-R Blockade
A Phase 2/3 study testing VAY736 in people with Autoimmune Hepatitis, run by Novartis Pharmaceuticals at 28 sites in 10 countries.
Trial summary
VAY736 dose testing; VAY736 efficacy and safety testing. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Autoimmune Hepatitis
- What is being tested?
- VAY736
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 68 participants
- Where
- 28 sitesin 10 countries
- Study dates
- 15 Feb 2018to 17 Dec 2025
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- AIH diagnosed per International Autoimmune Hepatitis Group
- Liver biopsy with Ishak modified HAI indicating active AIH
- Incomplete response to OR intolerance of standard therapy (per AASLD)
Who cannot join
- 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
- Required regular use of medications with known hepatotoxicity
- Decompensated cirrhosis
- Diagnosis of overlap syndrome with AIH (e.g., AIH+PBC, AIH+PSC).
- Drug related AIH at screening or a history of drug related AIH.
- History of drug abuse or unhealthy alcohol use
- History of malignancy of any organ system
- Pregnant or nursing (lactating) women
Trial locations and contacts
28 sites in 10 countries.
28 sites in 10 countries
Germany5 sites
Novartis Investigative Site
Aachen, 52074
Novartis Investigative Site
Hamburg, 20246
Novartis Investigative Site
Hanover, 30625
Novartis Investigative Site
Mainz, 55131
Novartis Investigative Site
Würzburg, Bavaria, 97080
United Kingdom5 sites
Novartis Investigative Site
Birmingham, West Midlands, B15 2TH
Novartis Investigative Site
London, SE5 9RS
Novartis Investigative Site
Newcastle upon Tyne, NE1 4LP
Novartis Investigative Site
Nottingham, NG7 2UH
Novartis Investigative Site
Oxford, OX3 9DU
United States4 sites
Southern California Research Center
Coronado, California, 92118
St. Lukes Advanced Liver Therapies
Houston, Texas, 77030
Inland Empire Liver Foundation
Rialto, California, 92377
University of California Davis Medical Center
Sacramento, California, 95817
Canada3 sites
Novartis Investigative Site
Edmonton, Alberta, T6G 2B7
Novartis Investigative Site
Montreal, Quebec, H2X 1R9
Novartis Investigative Site
Toronto, Ontario, M5G 2C4
Japan3 sites
Novartis Investigative Site
Itabashi Ku, Tokyo, 173 8606
Novartis Investigative Site
Sapporo, Hokkaido, 0068555
Novartis Investigative Site
Takamatsu, Kagawa-ken, 760 8557
Spain3 sites
Novartis Investigative Site
Barcelona, 08036
Novartis Investigative Site
Madrid, 28009
Novartis Investigative Site
Madrid, 28046
Argentina2 sites
Novartis Investigative Site
CABA, Buenos Aires, C1056ABI
Novartis Investigative Site
CABA, C1181ACH
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Study of the Safety and Efficacy of VAY736 in Autoimmune Hepatitis
VAY736 dose testing; VAY736 efficacy and safety testing.
Read the 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.
- Conditions
- Autoimmune Hepatitis
- Being tested
- VAY736, Placebo
- Keywords
- Type 1, Type 2, autoimmune hepatitis, AIH
- Medical subject headings
- Hepatitis, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VAY736Biological
VAY736
PlaceboOther
Placebo control with conversion to active VAY736
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | VAY736 Dose 1 Receives: Biological: VAY736 |
| Arm 2 | Experimental | VAY736 Dose 2 Receives: Biological: VAY736 |
| Arm 3 | Experimental | VAY736 Dose 3 Receives: Biological: VAY736 |
| Arm 4 | Placebo comparator | Placebo Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
ALT (Alanine aminotransferase) normalization
Difference in ALT normalization
Measured: Week 24
Secondary outcomes (1)
ALT normalization by dose
VAY736 dose-response
Measured: Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2017
- Study start15 Feb 2018
- Primary completion28 Dec 2023
- Study completion17 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Autoimmune HepatitisStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03217422
- Sponsor's protocol ID
- CVAY736B2201
- Other IDs
- 2023-508859-39-00, 2023
- Acronym
- AMBER
- Status last verified
- Feb 2026
- First submitted
- 12 Jul 2017
- Last updated
- 19 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Publications
Cholankeril G, Vierling JM. The clinical imperative of a complete biochemical response to immunosuppression in autoimmune hepatitis: Close is not good enough! Hepatology. 2024 Mar 1;79(3):529-531. doi: 10.1097/HEP.0000000000000597. Epub 2023 Sep 18. No abstract available.
PubMed 37722131Chung YY, Rahim MN, Heneghan MA. Autoimmune hepatitis and pregnancy: considerations for the clinician. Expert Rev Clin Immunol. 2022 Apr;18(4):325-333. doi: 10.1080/1744666X.2022.2044307. Epub 2022 Mar 2.
PubMed 35179437
Study at a glance
From the registry record, last updated by the sponsor on 19 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Study of the Safety and Efficacy of VAY736 in Autoimmune Hepatitis
- ClinicalTrials.gov ID
- NCT03217422
- Acronym / sponsor ID
- AMBER / CVAY736B2201
- Conditions
- Autoimmune Hepatitis
- Keywords
- Type 1, Type 2, autoimmune hepatitis, AIH
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 68 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 15 Feb 2018 (actual)
- Primary completion
- 28 Dec 2023 (actual)
- Study completion
- 17 Dec 2025 (actual)
- First posted
- 14 Jul 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Biological: VAY736VAY736 Dose 1 |
| Arm 2 | Experimental | Biological: VAY736VAY736 Dose 2 |
| Arm 3 | Experimental | Biological: VAY736VAY736 Dose 3 |
| Arm 4 | Placebo comparator | Other: PlaceboPlacebo |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- AIH diagnosed per International Autoimmune Hepatitis Group
- Liver biopsy with Ishak modified HAI indicating active AIH
- Incomplete response to OR intolerance of standard therapy (per AASLD)
Key exclusion criteria (8 listed)
- 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
- Required regular use of medications with known hepatotoxicity
- Decompensated cirrhosis
- Diagnosis of overlap syndrome with AIH (e.g., AIH+PBC, AIH+PSC).
- Drug related AIH at screening or a history of drug related AIH.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VAY736VAY736 | Biological | Arm 1, Arm 2, Arm 3 |
| PlaceboPlacebo control with conversion to active VAY736 | Other | Arm 4 |
Primary outcome
ALT (Alanine aminotransferase) normalization
Time frame: Week 24
Difference in ALT normalization
1 secondary outcome
ALT normalization by dose
Time frame: Week 24
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Feb 2026.
- Derived from this study: PubMed 37722131Cholankeril G, Vierling JM. The clinical imperative of a complete biochemical response to immunosuppression in autoimmune hepatitis: Close is not good enough! Hepatology. 2024 Mar 1;79(3):529-531. doi: 10.1097/HEP.0000000000000597. Epub 2023 Sep 18. No abstract available.
- Derived from this study: PubMed 35179437Chung YY, Rahim MN, Heneghan MA. Autoimmune hepatitis and pregnancy: considerations for the clinician. Expert Rev Clin Immunol. 2022 Apr;18(4):325-333. doi: 10.1080/1744666X.2022.2044307. Epub 2022 Mar 2.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Novartis Pharmaceuticals
28 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing VAY736 in people with Autoimmune Hepatitis, run by Novartis Pharmaceuticals at 28 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in Germany, United Kingdom, United States, Canada, Japan and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ALT (Alanine aminotransferase) normalization (time frame: Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03217422, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Feb 2026.