ADCC Mediated B-Cell dEpletion and BAFF-R Blockade

Phase 2

Active, not recruiting

• Conditions: Autoimmune Hepatitis
• Interventions: Biological: VAY736; Other: Placebo

• Registration Number: NCT03217422
• Lead Sponsor: Novartis Pharmaceuticals

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.

Study Timeline

• Study First Submitted: 2017-07-12
• Study First Submitted QC: 2017-07-12
• Study First Posted: 2017-07-14
• Study Start: 2018-02-15
• Primary Completion: 2023-12-28
• Disposition First Posted: 2024-09-19
• Disposition First Submitted: 2024-11-25
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-19
• Last Update Posted: 2024-12-24
• Study Completion: 2025-12-25

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 68

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

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Arm 1	VAY736	VAY736 Dose 1
Arm 2	VAY736	VAY736 Dose 2
Arm 4	Placebo	Placebo
Arm 3	VAY736	VAY736 Dose 3

Primary Outcome Measures

Name	Time	Method
ALT (Alanine aminotransferase) normalization	Week 24	Difference in ALT normalization

Secondary Outcome Measures

Name	Time	Method
ALT normalization by dose	Week 24	VAY736 dose-response

Trial Locations

Locations (5)

Southern California Research Center; 🇺🇸 Coronado, California, United States

Inland Empire Liver Foundation; 🇺🇸 Rialto, California, United States

University of California Davis Medical Center; 🇺🇸 Sacramento, California, United States

St. Lukes Advanced Liver Therapies; 🇺🇸 Houston, Texas, United States

Novartis Investigative Site; 🇬🇧 Oxford, United Kingdom