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

  • End date
    Feb 13, 2029
  • participants needed
  • sponsor
    Novartis Pharmaceuticals
Updated on 5 April 2023


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.


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.

Condition Warm Autoimmune Hemolytic Anemia (wAIHA)
Treatment Placebo, ianalumab
Clinical Study IdentifierNCT05648968
SponsorNovartis Pharmaceuticals
Last Modified on5 April 2023


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Inclusion Criteria

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 <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 other immunologic disease requiring immunosuppressant treatments that are not allowed in this study
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
Neutrophils: <1000/mm3
Serum creatinine >1.5 × upper limit of normal (ULN) for adults or estimated glomerular filtration rate (eGFR)≤60 mL/min for adolescent
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)
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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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