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Evaluating the effectiveness of ianalumab for treating warm autoimmune hemolytic anemia

A Phase 3, Randomized, Double-blind, Study to Assess Efficacy and Safety of Ianalumab (VAY736) Versus Placebo in Warm Autoimmune Hemolytic Anemia (wAIHA) Patients Who Failed at Least One Line of Treatment

PHASE3 · Novartis · NCT05648968

This study is testing if a new drug called ianalumab can help people with warm autoimmune hemolytic anemia who haven't found relief from other treatments.

Quick facts

PhasePHASE3
Study typeInterventional
Enrollment90 (estimated)
Ages18 Years to 100 Years
SexAll
SponsorNovartis (industry)
Drugs / interventionsrituximab, ianalumab
Locations89 sites (Aurora, Colorado and 88 other locations)
Trial IDNCT05648968 on ClinicalTrials.gov

What this trial studies

This study aims to assess the efficacy and safety of ianalumab in patients with warm autoimmune hemolytic anemia (wAIHA) who have not responded to previous treatments. Participants will be randomly assigned to receive either ianalumab at two different doses or a placebo. The primary goal is to demonstrate that ianalumab can induce a durable hemoglobin response compared to placebo. Additionally, the study will monitor the maintenance of this response beyond the treatment period, with follow-up visits scheduled throughout the study duration.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with documented wAIHA who have had an insufficient response to or relapsed after at least one line of treatment.

Not a fit: Patients with wAIHA secondary to hematologic diseases or those who have previously used B-cell depleting therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for patients with wAIHA who have not responded to existing therapies.

How similar studies have performed: While this approach is novel in the context of wAIHA, similar studies targeting B-cell depletion have shown promise in other autoimmune conditions.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key 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

Key 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.

Where this trial is running

Aurora, Colorado and 88 other locations

+39 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Warm Autoimmune Hemolytic Anemia, warm autoimmune hemolytic anemia, wAIHA, ianalumab, VAY736, B-cell depletion, B-cell Activating Factor Receptor blockade

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.