A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia

NCT05648968 | Active Not Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: CVAY736O123012022-001773-312024-510635-21-00
• Study Type: interventional
• Target: 90
• Locations: 16 countries
• Sites: 53

Brief Summary

The purpose of this study is to evaluate efficacy and safety of ianalumab compared to placebo in patients with warm autoimmune hemolytic anemia, who failed at least one line of treatment.

Conditions

Warm Autoimmune Hemolytic Anemia (wAIHA)

Keywords

warm autoimmune hemolytic anemia; wAIHA; ianalumab; VAY736; B-cell depletion; B-cell Activating Factor Receptor (BAFF-R) blockade

Outcome Measures

Primary Outcomes (1)

• Binary variable indicating whether a patient achieves a durable response

  Durable response: hemoglobin level ≥10 g/dL and ≥2 g/dL increase from baseline, for a period of at least eight consecutive weeks between W9 and W25, in the absence of rescue medication or prohibited treatment

  Randomization to Week 25

Secondary Outcomes (20)

• Duration of response (Key Secondary)

  Randomization to end of study (up to 39 months after randomization of last patient)

• Time from randomization to start of durable response in each treatment group

  Randomization to end of study (up to 39 months after randomization of last patient)

• Time from randomization to start of first response in each treatment group

  Randomization to end of study (up to 39 months after randomization of last patient)

• Time from randomization to start of complete response in each treatment group

  Randomization to end of study (up to 39 months after randomization of last patient)

• Response rate

  Randomization to end of study (up to 39 months after randomization of last patient)

Study Arms (3)

Ianalumab low dose

EXPERIMENTAL

Participants will receive low dose ianalumab intravenously

Biological: Ianalumab

Ianalumab high dose

EXPERIMENTAL

Participants will receive high dose ianalumab intravenously

Biological: Ianalumab

Placebo

PLACEBO COMPARATOR

Participants will receive placebo intravenously

Drug: Placebo

Interventions

Ianalumab BIOLOGICAL

i.v. infusion, prepared from concentrate solution

Also known as: VAY736

Ianalumab high dose; Ianalumab low dose

Placebo DRUG

i.v. infusion, prepared from matching placebo

Placebo

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• years and older at time of signing consent
• Patients with primary or secondary wAIHA documented by positive direct antiglobulin test specific for anti-IgG or anti-IgA, who had an insufficient response to, or relapsed after at least one line of treatment, including patients with steroid resistance, dependence or intolerance
• Hemoglobin concentration at screening and at Week 1 \>=5 g/dL and \<10 g/dL, associated with presence of symptoms related to anemia
• The dose of supportive care must be stable for at least 4 weeks prior to randomization into the study

You may not qualify if:

• wAIHA secondary to hematologic disease involving bone marrow (e.g., CLL) or another immunologic disease requiring prohibited medication as per protocol. Patients with autoimmune diseases after wash-out from the treatments are allowed.
• Presence of other forms of AIHA (cold or intermediate forms), Evans Syndrome or other cytopenias
• Prior use of B-cell depleting therapy (e.g., rituximab) within 12 weeks prior to randomization, or without hematological response to the last course of B-cell depleting therapy
• Neutrophils: \<1000/mm3
• Serum creatinine \>1.5 × upper limit of normal (ULN)
• Immunoglobulin G (IgG) \<5g/L
• Active viral, bacterial or other infections (including tuberculosis and SARS-CoV-2) requiring systemic treatment at time of screening, or history of recurrent clinically significant infection
• Positivity for hepatitis C virus, hepatitis B surface antigen (HBsAg), or hepatitis B core antibody (HBcAb). HBcAb positive patients can be enrolled if HBsAg negative, HBV DNA negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given.
• Known history of primary or secondary immunodeficiency, or a positive human immune deficiency virus (HIV) test result
• Live or live-attenuated vaccination within 4 weeks before randomization
• History of splenectomy

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (53)

Michigan Center of Medical Research

Farmington Hills, Michigan, 48334, United States

Location

University of Minnesota Med Center

Minneapolis, Minnesota, 55455, United States

Location

Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States

Location

Novartis Investigative Site

CABA, Buenos Aires, C1414DRK, Argentina

Location

Novartis Investigative Site

Caba, C1015ABO, Argentina

Location

Novartis Investigative Site

Caba, C1039AAC, Argentina

Location

Novartis Investigative Site

Garran, Australian Capital Territory, 2605, Australia

Location

Novartis Investigative Site

Melbourne, Victoria, 3004, Australia

Location

Novartis Investigative Site

Guangzhou, Guangdong, 510515, China

Location

Novartis Investigative Site

Hangzhou, Zhejiang, 310003, China

Location

Novartis Investigative Site

Dalian, 116000, China

Location

Novartis Investigative Site

Tianjin, 300020, China

Location

Novartis Investigative Site

Tianjin, 300052, China

Location

Novartis Investigative Site

Créteil, 94010, France

Location

Novartis Investigative Site

Lille, 59037, France

Location

Novartis Investigative Site

Nantes, 44093, France

Location

Novartis Investigative Site

Nice, 06202, France

Location

Novartis Investigative Site

Dresden, 01307, Germany

Location

Novartis Investigative Site

Essen, 45147, Germany

Location

Novartis Investigative Site

Giessen, 35392, Germany

Location

Novartis Investigative Site

Hanover, 30161, Germany

Location

Novartis Investigative Site

Debrecen, Hajdu Bihar Megye, 4032, Hungary

Location

Novartis Investigative Site

New Delhi, National Capital Territory of Delhi, 110029, India

Location

Novartis Investigative Site

Madurai, Tamil Nadu, 625107, India

Location

Novartis Investigative Site

Hyderabad, Telangana, 500082, India

Location

Novartis Investigative Site

Lucknow, Uttar Pradesh, 226014, India

Location

Novartis Investigative Site

Kfar Saba, 4428164, Israel

Location

Novartis Investigative Site

Petah Tikva, 4941492, Israel

Location

Novartis Investigative Site

Milan, MI, 20100, Italy

Location

Novartis Investigative Site

Milan, MI, 20122, Italy

Location

Novartis Investigative Site

Bassano del Grappa, VI, 36061, Italy

Location

Novartis Investigative Site

Novara, 28100, Italy

Location

Novartis Investigative Site

Narita, Chiba, 286-8523, Japan

Location

Novartis Investigative Site

Matsuyama, Ehime, 7900024, Japan

Location

Novartis Investigative Site

Gifu, Gifu, 501-1194, Japan

Location

Novartis Investigative Site

Kobe, Hyōgo, 6500047, Japan

Location

Novartis Investigative Site

Isehara, Kanagawa, 259-1193, Japan

Location

Novartis Investigative Site

Suita, Osaka, 5650871, Japan

Location

Novartis Investigative Site

Itabashi-ku, Tokyo, 1738610, Japan

Location

Novartis Investigative Site

Shinjuku Ku, Tokyo, 160-0023, Japan

Location

Novartis Investigative Site

George Town, Pulau Pinang, 10450, Malaysia

Location

Novartis Investigative Site

Kuching, Sarawak, 93586, Malaysia

Location

Novartis Investigative Site

Johor Bahru, 80100, Malaysia

Location

Novartis Investigative Site

Singapore, 119074, Singapore

Location

Novartis Investigative Site

Singapore, S308433, Singapore

Location

Novartis Investigative Site

Barcelona, 08035, Spain

Location

Novartis Investigative Site

Murcia, 30008, Spain

Location

Novartis Investigative Site

Bangkok, 10330, Thailand

Location

Novartis Investigative Site

Bangkok, 10700, Thailand

Location

Novartis Investigative Site

Chiang Mai, 50200, Thailand

Location

Novartis Investigative Site

Birmingham, West Midlands, B15 2TH, United Kingdom

Location

Novartis Investigative Site

Leeds, LS1 3EX, United Kingdom

Location

Novartis Investigative Site

London, W12 0HS, United Kingdom

Location

MeSH Terms

Interventions

ianalumab

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: November 23, 2022
• First Posted: December 13, 2022
• Study Start: December 30, 2022
• Primary Completion: February 20, 2026
• Study Completion (Estimated): December 19, 2028
• Last Updated: March 19, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

IN (4); IL (2); DE (4); US (3); IT (4); GB (3); HU (1); ES (2); JP (8); AU (2); TH (3); AR (3); FR (4); CN (5); MY (3); SG (2)