Last Update: Mar 25, 2024
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:
Novartis Reference Number:CVAY736O12301
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. 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.

Warm Autoimmune Hemolytic Anemia (wAIHA)
Phase3
Recruiting
90
Dec 30, 2022
Feb 08, 2029
All
18 Years - (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 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.

Study Location

Novartis Investigative Site

Recruiting

Ciudad Autonoma de Bs As,Buenos Aires,C1015abo,Argentina

Novartis Investigative Site

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Buenos aires,C1039aac,Argentina

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Caba,Buenos Aires,C1414drk,Argentina

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Prahran,Victoria,3181,Australia

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Canberra,Australian Capital Territory,2605,Australia

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Hangzhou,Zhejiang,310003,China

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Tianjin,300052,China

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Kunming,Yunnan,650101,China

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Dalian,116000,China

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Wuhan,Hubei,430022,China

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Suzhou,Jiangsu,215006,China

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Guangzhou,Guangdong,510515,China

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Tianjin,300020,China

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Blois Cedex,41000,France

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Toulouse,31059,France

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Creteil,94010,France

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Nantes Cedex 1,44093,France

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Vandoeuvre Les Nancy,54511,France

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Le Mans,Cedex 09,72037,France

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Caen,Cedex,14033,France

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Lille Cedex,59 037,France

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Nice,06202,France

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Frankfurt,60590,Germany

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Hannover,30161,Germany

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Giessen,35392,Germany

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Greifswald,17475,Germany

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Dresden,01307,Germany

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Essen,45147,Germany

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Debrecen,4032,Hungary

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Lucknow,Uttar Pradesh,226014,India

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Madurai,Tamil NADU,625107,India

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New Delhi,110029,India

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Petach Tikva,4941492,Israel

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Afula,1834111,Israel

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Kfar Saba,44281,Israel

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Zerifin,HaMerkaz,7030000,Israel

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Milano,MI,20157,Italy

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Novara,28100,Italy

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Avellino,AV,83100,Italy

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Bari,BA,70124,Italy

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Milano,MI,20122,Italy

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Bassano Del Grappa,VI,36061,Italy

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Shinjuku-ku,Tokyo,160-0023,Japan

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Fukuoka city,Fukuoka,812-8582,Japan

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Isehara,Kanagawa,259-1193,Japan

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Itabashi-ku,Tokyo,173-8610,Japan

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Suita,Osaka,565 0871,Japan

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Gifu-city,Gifu,501-1194,Japan

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Narita,Chiba,286-8523,Japan

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Matsuyama-city,Ehime,790-0024,Japan

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Yamagata,990 9585,Japan

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Aomori,030 8553,Japan

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Kobe-city,Hyogo,650-0047,Japan

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Kuala Lumpur,MYS,56000,Malaysia

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Penang,10050,Malaysia

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Kuching,Sarawak,93586,Malaysia

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Johor Bahru,80100,Malaysia

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Pulau Pinang,10990,Malaysia

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Selangor,68000,Malaysia

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Bucuresti,013975,Romania

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Singapore,119228,Singapore

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Singapore,S308433,Singapore

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Murcia,30008,Spain

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Barcelona,Catalunya,08035,Spain

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Taoyuan,33305,Taiwan

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Chiang Mai,50200,Thailand

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Bangkok,10330,Thailand

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Bangkok,10700,Thailand

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London,E1 1bb,United Kingdom

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London,W12 0hs,United Kingdom

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Leeds,Ls1 3ex,United Kingdom

Inspira Medical Cent Mullica Hill

Recruiting

Mullica Hill,New Jersey,08062,United States

Jennifer Hart
Erev Tubb

STAT Research Inc Premier Clin Res LLC STAT Res

Recruiting

Dayton,Ohio,45402,United States

Charles Bane

Montefiore Medical Center .

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Bronx,New York,10461,United States

Irina Murakhovskaya
Noelle Townsend

University of Minnesota Med Center

Recruiting

Minneapolis,Minnesota,55455,United States

Diondra Howard
Marshall Mazepa

Napa Research

Recruiting

Margate,Florida,33063,United States

Emilio Araujo-Mino
Maria Marin

Gabrail Cancer Center

Recruiting

Canton,Ohio,44718,United States

Nashat Y Gabrail

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