A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With 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

ClinicalTrials.gov Identifier: NCT05648968

Novartis Reference Number: CVAY736O12301

Last Update: Jan 09, 2023

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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.

Condition

Warm Autoimmune Hemolytic Anemia (wAIHA)

Phase

Phase 3

Overall status

Recruiting

Start date

Dec 30, 2022

Completion date

Feb 13, 2029

Gender

All

Age(s)

18 Years and older (Adult, Older Adult)

Interventions

Biological

Ianalumab

i.v. infusion, prepared from concentrate solution

Drug

Placebo

i.v. infusion, prepared from matching placebo

Eligibility 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 <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 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.