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.
Interventions
Ianalumab
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.
Novartis Investigative Site
Recruiting
Caba,Buenos Aires,C1414drk,Argentina
Novartis Investigative Site
Recruiting
Ciudad Autonoma de Bs As,Buenos Aires,C1015abo,Argentina
Novartis Investigative Site
Recruiting
Buenos aires,C1039aac,Argentina
Novartis Investigative Site
Recruiting
Canberra,Australian Capital Territory,2605,Australia
Novartis Investigative Site
Recruiting
Melbourne,Victoria,3004,Australia
Novartis Investigative Site
Recruiting
Dalian,116000,China
Novartis Investigative Site
Recruiting
Tianjin,300020,China
Novartis Investigative Site
Recruiting
Guangzhou,Guangdong,510515,China
Novartis Investigative Site
Recruiting
Tianjin,300052,China
Novartis Investigative Site
Recruiting
Wuhan,Hubei,430022,China
Novartis Investigative Site
Recruiting
Suzhou,Jiangsu,215004,China
Novartis Investigative Site
Recruiting
Kunming,Yunnan,650101,China
Novartis Investigative Site
Recruiting
Hangzhou,Zhejiang,310003,China
Novartis Investigative Site
Recruiting
Le Mans,72000,France
Novartis Investigative Site
Recruiting
Lille,59037,France
Novartis Investigative Site
Recruiting
Nantes Cedex 1,44093,France
Novartis Investigative Site
Recruiting
Nice,06202,France
Novartis Investigative Site
Recruiting
Blois Cedex,41000,France
Novartis Investigative Site
Recruiting
Toulouse,31059,France
Novartis Investigative Site
Recruiting
Caen,14033,France
Novartis Investigative Site
Recruiting
Vandoeuvre Les Nancy,54511,France
Novartis Investigative Site
Recruiting
Creteil,94010,France
Novartis Investigative Site
Recruiting
Essen,45147,Germany
Novartis Investigative Site
Recruiting
Frankfurt,60590,Germany
Novartis Investigative Site
Recruiting
Giessen,35392,Germany
Novartis Investigative Site
Recruiting
Hannover,30161,Germany
Novartis Investigative Site
Recruiting
Dresden,01307,Germany
Novartis Investigative Site
Recruiting
Debrecen,4032,Hungary
Novartis Investigative Site
Recruiting
Hyderabad,Telangana,500082,India
Novartis Investigative Site
Recruiting
Lucknow,Uttar Pradesh,226014,India
Novartis Investigative Site
Recruiting
New Delhi,110029,India
Novartis Investigative Site
Recruiting
Madurai,TamilNadu,625107,India
Novartis Investigative Site
Recruiting
Petach Tikva,4941492,Israel
Novartis Investigative Site
Recruiting
Zerifin,7030000,Israel
Novartis Investigative Site
Recruiting
Afula,1834111,Israel
Novartis Investigative Site
Recruiting
Kfar Saba,44281,Israel
Novartis Investigative Site
Recruiting
Novara,28100,Italy
Novartis Investigative Site
Recruiting
Avellino,AV,83100,Italy
Novartis Investigative Site
Recruiting
Bari,BA,70124,Italy
Novartis Investigative Site
Recruiting
Milano,MI,20100,Italy
Novartis Investigative Site
Recruiting
Milano,MI,20122,Italy
Novartis Investigative Site
Recruiting
Bassano Del Grappa,Vicenza,36061,Italy
Novartis Investigative Site
Recruiting
Isehara,Kanagawa,259-1193,Japan
Novartis Investigative Site
Recruiting
Suita,Osaka,565 0871,Japan
Novartis Investigative Site
Recruiting
Narita,Chiba,286-8523,Japan
Novartis Investigative Site
Recruiting
Itabashi-ku,Tokyo,173-8610,Japan
Novartis Investigative Site
Recruiting
Matsuyama-city,Ehime,790-0024,Japan
Novartis Investigative Site
Recruiting
Shinjuku Ku,Tokyo,160-0023,Japan
Novartis Investigative Site
Recruiting
Fukuoka city,Fukuoka,812-8582,Japan
Novartis Investigative Site
Recruiting
Aomori,030 8553,Japan
Novartis Investigative Site
Recruiting
Gifu-city,Gifu,501-1194,Japan
Novartis Investigative Site
Recruiting
Yamagata,990 9585,Japan
Novartis Investigative Site
Recruiting
Kobe,Hyogo,650-0047,Japan
Novartis Investigative Site
Recruiting
Johor Bahru,80100,Malaysia
Novartis Investigative Site
Recruiting
Kuala Lumpur,56000,Malaysia
Novartis Investigative Site
Recruiting
Penang,10050,Malaysia
Novartis Investigative Site
Recruiting
Pulau Pinang,10990,Malaysia
Novartis Investigative Site
Recruiting
Selangor,68000,Malaysia
Novartis Investigative Site
Recruiting
Kuching,Sarawak,93586,Malaysia
Novartis Investigative Site
Recruiting
Bucuresti,013975,Romania
Novartis Investigative Site
Recruiting
Singapore,119074,Singapore
Novartis Investigative Site
Recruiting
Singapore,S308433,Singapore
Novartis Investigative Site
Recruiting
Barcelona,Catalunya,08035,Spain
Novartis Investigative Site
Recruiting
Murcia,30008,Spain
Novartis Investigative Site
Recruiting
Taoyuan,33305,Taiwan
Novartis Investigative Site
Recruiting
Bangkok,10700,Thailand
Novartis Investigative Site
Recruiting
Chiang Mai,50200,Thailand
Novartis Investigative Site
Recruiting
Bangkok,10330,Thailand
Novartis Investigative Site
Recruiting
Birmingham,B15 2th,United Kingdom
Novartis Investigative Site
Recruiting
Leeds,Ls1 3ex,United Kingdom
Novartis Investigative Site
Recruiting
London,E1 1bb,United Kingdom
Novartis Investigative Site
Recruiting
London,W12 0hs,United Kingdom
Michigan Center of Medical Research
Recruiting
Farmington Hills,Michigan,48334,United States
Faisal Musa
Texas Oncology-Baylor Scott and White
Recruiting
Dallas,Texas,75231,United States
Moshe Yair Levy
University of Colorado Anschutz
Recruiting
Aurora,Colorado,80045,United States
George Gemlyn
Montefiore Medical Center .
Recruiting
Bronx,New York,10461,United States
Irina Murakhovskaya
University of Minnesota Med Center
Recruiting
Minneapolis,Minnesota,55455,United States
Diondra Howard
Marshall Mazepa
Fred Hutchinson Cancer Center
Recruiting
Seattle,Washington,98109,United States
Sandhya Panch
Napa Research
Recruiting
Margate,Florida,33063,United States
Emilio Araujo-Mino
Maria Marin
NorthShore University Health System
Recruiting
Evanston,Illinois,60201,United States
Amy Wang
STAT Research Inc Premier Clin Res LLC STAT Res
Recruiting
Dayton,Ohio,45402,United States
Charles Bane
Inspira Medical Cent Mullica Hill
Recruiting
Mullica Hill,New Jersey,08062,United States
Erev Tubb
Parkview Research Center .
Recruiting
Fort Wayne,Indiana,46845,United States
Jeffrey Letzer
Worldwide Contacts
If the location of your choosing does not feature any contact detail, please reach out using the information below.