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

NCT ID: NCT05648968

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

90 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-12-30

Study Completion Date

2029-05-02

Brief Summary

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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.

Detailed Description

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The primary objective is to demonstrate that either dose of ianalumab induces a durable hemoglobin response compared to placebo in patients with wAIHA.

The key secondary objective is to demonstrate that either dose of ianalumab maintains a durable hemoglobin response that is sustained beyond end of the treatment period, compared to placebo.

Participants are randomized to two different doses of ianalumab or placebo. Participants who were assigned to placebo arm and not responding to treatment may be treated with open label ianalumab using the higher dose.

The investigational treatment will be supplied in a double-blinded manner. For the open label period, ianalumab will be provided in an open label manner.

In addition to the randomized treatment (ianalumab or placebo), specific supportive care medication as defined in the protocol is allowed. If clinically indicated (e.g., to ensure patient safety), the treating physician may also administer rescue medication.

The study consists of the treatment period, efficacy and safety follow-up periods. The visit frequency will be every other week during the treatment and primary endpoint follow up period; for safety monitoring monthly during the first 20 weeks after last dose and afterwards quarterly up to 2 years from the last dose. For participants in durable response, additional visits for efficacy will occur monthly during the first 2 years after the last dose, and afterwards quarterly until loss of response or end of study, latest until up to 39 months post randomization of the last participant.

Conditions

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Warm Autoimmune Hemolytic Anemia (wAIHA)

Keywords

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Ianalumab low dose

Participants will receive low dose ianalumab intravenously

Group Type EXPERIMENTAL

Ianalumab

Intervention Type BIOLOGICAL

i.v. infusion, prepared from concentrate solution

Ianalumab high dose

Participants will receive high dose ianalumab intravenously

Group Type EXPERIMENTAL

Ianalumab

Intervention Type BIOLOGICAL

i.v. infusion, prepared from concentrate solution

Placebo

Participants will receive placebo intravenously

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

i.v. infusion, prepared from matching placebo

Interventions

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Ianalumab

i.v. infusion, prepared from concentrate solution

Intervention Type BIOLOGICAL

Placebo

i.v. infusion, prepared from matching placebo

Intervention Type DRUG

Other Intervention Names

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VAY736

Eligibility Criteria

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Inclusion Criteria

* 18 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

Exclusion Criteria

* 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

Other protocol-defined Inclusion/Exclusion may apply.
Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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University of Colorado Anschutz

Aurora, Colorado, United States

Site Status WITHDRAWN

Napa Research

Margate, Florida, United States

Site Status WITHDRAWN

NorthShore University Health System

Evanston, Illinois, United States

Site Status WITHDRAWN

Parkview Research Center

Fort Wayne, Indiana, United States

Site Status WITHDRAWN

Michigan Center of Medical Research

Farmington Hills, Michigan, United States

Site Status ACTIVE_NOT_RECRUITING

University of Minnesota Med Center

Minneapolis, Minnesota, United States

Site Status ACTIVE_NOT_RECRUITING

Summit Health

Florham Park, New Jersey, United States

Site Status WITHDRAWN

Inspira Medical Cent Mullica Hill

Mullica Hill, New Jersey, United States

Site Status WITHDRAWN

Montefiore Medical Center

The Bronx, New York, United States

Site Status WITHDRAWN

Brody School of Medicine

Greenville, North Carolina, United States

Site Status WITHDRAWN

Gabrail Cancer Center

Canton, Ohio, United States

Site Status RECRUITING

STAT Research Inc

Dayton, Ohio, United States

Site Status WITHDRAWN

Texas Oncology-Baylor Scott and White

Dallas, Texas, United States

Site Status WITHDRAWN

Baylor College Of Medicine

Houston, Texas, United States

Site Status WITHDRAWN

Fred Hutchinson Cancer Center

Seattle, Washington, United States

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

CABA, , Argentina

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Caba, , Argentina

Site Status RECRUITING

Novartis Investigative Site

Garran, Australian Capital Territory, Australia

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Fitzroy, Victoria, Australia

Site Status RECRUITING

Novartis Investigative Site

Melbourne, Victoria, Australia

Site Status RECRUITING

Novartis Investigative Site

Guangzhou, Guangdong, China

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Wuhan, Hubei, China

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Suzhou, Jiangsu, China

Site Status WITHDRAWN

Novartis Investigative Site

Tianjin, Tianjin Municipality, China

Site Status RECRUITING

Novartis Investigative Site

Kunming, Yunnan, China

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Hangzhou, Zhejiang, China

Site Status COMPLETED

Novartis Investigative Site

Dalian, , China

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Tianjin, , China

Site Status RECRUITING

Novartis Investigative Site

Blois, , France

Site Status WITHDRAWN

Novartis Investigative Site

Caen, , France

Site Status WITHDRAWN

Novartis Investigative Site

Créteil, , France

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Le Mans, , France

Site Status WITHDRAWN

Novartis Investigative Site

Lille, , France

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Nantes, , France

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Nice, , France

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Toulouse, , France

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Vandœuvre-lès-Nancy, , France

Site Status WITHDRAWN

Novartis Investigative Site

Frankfurt am Main, Hesse, Germany

Site Status RECRUITING

Novartis Investigative Site

Dresden, , Germany

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Essen, , Germany

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Giessen, , Germany

Site Status COMPLETED

Novartis Investigative Site

Greifswald, , Germany

Site Status WITHDRAWN

Novartis Investigative Site

Hanover, , Germany

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Debrecen, Hajdu Bihar Megye, Hungary

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Madurai, Tamil Nadu, India

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Hyderabad, Telangana, India

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Lucknow, Uttar Pradesh, India

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

New Delhi, , India

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Afula, , Israel

Site Status WITHDRAWN

Novartis Investigative Site

Kfar Saba, , Israel

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Petah Tikva, , Israel

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Ẕerifin, , Israel

Site Status WITHDRAWN

Novartis Investigative Site

Avellino, AV, Italy

Site Status RECRUITING

Novartis Investigative Site

Bari, BA, Italy

Site Status RECRUITING

Novartis Investigative Site

Milan, MI, Italy

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Milan, MI, Italy

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Bassano del Grappa, VI, Italy

Site Status RECRUITING

Novartis Investigative Site

Novara, , Italy

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Narita, Chiba, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Matsuyama, Ehime, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Fukuoka, Fukuoka, Japan

Site Status WITHDRAWN

Novartis Investigative Site

Gifu, Gifu, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Kobe, Hyōgo, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Isehara, Kanagawa, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Suita, Osaka, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Itabashi-ku, Tokyo, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Shinjuku Ku, Tokyo, Japan

Site Status COMPLETED

Novartis Investigative Site

Aomori, , Japan

Site Status WITHDRAWN

Novartis Investigative Site

Yamagata, , Japan

Site Status WITHDRAWN

Novartis Investigative Site

George Town, Pulau Pinang, Malaysia

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Kuching, Sarawak, Malaysia

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

George Town, , Malaysia

Site Status WITHDRAWN

Novartis Investigative Site

Johor Bahru, , Malaysia

Site Status COMPLETED

Novartis Investigative Site

Kuala Lumpur, , Malaysia

Site Status WITHDRAWN

Novartis Investigative Site

Kuala Selangor, , Malaysia

Site Status WITHDRAWN

Novartis Investigative Site

Bucharest, , Romania

Site Status WITHDRAWN

Novartis Investigative Site

Singapore, , Singapore

Site Status COMPLETED

Novartis Investigative Site

Singapore, , Singapore

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Barcelona, Catalonia, Spain

Site Status RECRUITING

Novartis Investigative Site

Murcia, , Spain

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Taoyuan District, , Taiwan

Site Status WITHDRAWN

Novartis Investigative Site

Bangkok, , Thailand

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Bangkok, , Thailand

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Chiang Mai, , Thailand

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Birmingham, , United Kingdom

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Leeds, , United Kingdom

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

London, , United Kingdom

Site Status RECRUITING

Novartis Investigative Site

London, , United Kingdom

Site Status ACTIVE_NOT_RECRUITING

Countries

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United States Argentina Australia China France Germany Hungary India Israel Italy Japan Malaysia Romania Singapore Spain Taiwan Thailand United Kingdom

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

Phone: 1-888-669-6682

Email: [email protected]

Novartis Pharmaceuticals

Role: CONTACT

Phone: +41613241111

Facility Contacts

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Role: primary

Other Identifiers

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2022-001773-31

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2024-510635-21-00

Identifier Type: OTHER

Identifier Source: secondary_id

CVAY736O12301

Identifier Type: -

Identifier Source: org_study_id