A study of efficacy and safety of ianalumab in previously treated patients with warm autoimmune hemolytic anemia
Sponsor(s) :
Novartis Pharmaceuticals
Recruiment : closed
Participating centers
21
Last modification : 2026-06-07
STUDY DESCRIPTION
Study summary
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.
Source : Import from center
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.
Source : Import from center
RECRUITMENT
Participant profile
Age limits
minimum: 18 years old
maximum: 100 years old
Participants gender(s)
ALL
Source : Import from center
Medical condition (targeted specialty)
Research domain
Data not available
Selection criterias
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.
Cohorts
Name
Medical condition
Treatment
Recruitment status
Ianalumab low dose
Participants will receive low dose ianalumab intravenously
Data not available
Unknown
Ianalumab high dose
Participants will receive high dose ianalumab intravenously
Data not available
Unknown
Placebo
Participants will receive placebo intravenously
Data not available
Unknown
Ianalumab low dose
Recruitment status
unknown
Participants will receive low dose ianalumab intravenously
Ianalumab high dose
Recruitment status
unknown
Participants will receive high dose ianalumab intravenously
Placebo
Recruitment status
unknown
Participants will receive placebo intravenously
Current data since :
June 07, 2026 20:00
LOCATIONS AND CONTACTS
Main location
novartis investigative site
CONNOR DOWNS, CORNWALL, UNITED KINGDOM
Local recruitment
—
CLOSED
Also available at: MINNEAPOLIS, (MINNESOTA), GUANGZHOU, (GUANGDONG), SEATTLE, (WASHINGTON), GRANDERO BAIGORRIA, (SANTA FE), CHRISTCHURCH, (VICTORIA), BENGBU, (JIANGSU), ANGERS CEDEX 1, (COTE D OR), KIRCHHEIM UNTER TECK, (BADEN-WUERTTEMBERG), HATVAN, (BARANYA), SECUNDERABAD, (TELANGANA), HYDERABAD, (TELANGANA), ...
and 9 other cities.
Last modification :
June 07, 2026
Current data since :
09 Jun 10:18
Data source :
clinicaltrials.gov
* 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.
Source : Import from center
Cohorts
Proposed Therapy or Intervention
Cohorts
Name
Medical condition
Treatment
Recruitment status
Ianalumab low dose
Participants will receive low dose ianalumab intravenously
Data not available
Unknown
Ianalumab high dose
Participants will receive high dose ianalumab intravenously
Data not available
Unknown
Placebo
Participants will receive placebo intravenously
Data not available
Unknown
Ianalumab low dose
Recruitment status
unknown
Participants will receive low dose ianalumab intravenously
Ianalumab high dose
Recruitment status
unknown
Participants will receive high dose ianalumab intravenously
Placebo
Recruitment status
unknown
Participants will receive placebo intravenously
Current data since :
June 07, 2026 20:00
Study's description
Study description
Study summary
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.
Source : Import from center
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.
Source : Import from center
Locations
Locations
Participating centers
10
shown
out of
21
centers
FRED HUTCHINSON CANCER CENTER
Seattle
WASHINGTON, UNITED STATES
Local recruitment
Recruiting status:
CLOSED
NOVARTIS INVESTIGATIVE SITE
Bengbu
JIANGSU, CHINA
Local recruitment
Recruiting status:
CLOSED
NOVARTIS INVESTIGATIVE SITE
Grandero baigorria
SANTA FE, ARGENTINA
Local recruitment
Recruiting status:
CLOSED
NOVARTIS INVESTIGATIVE SITE
Christchurch
VICTORIA, AUSTRALIA
Local recruitment
Recruiting status:
CLOSED
NOVARTIS INVESTIGATIVE SITE
Guangzhou
GUANGDONG, CHINA
Local recruitment
Recruiting status:
CLOSED
NOVARTIS INVESTIGATIVE SITE
Angers cedex 1
COTE D OR, FRANCE
Local recruitment
Recruiting status:
CLOSED
NOVARTIS INVESTIGATIVE SITE
Kirchheim unter teck
BADEN-WUERTTEMBERG, GERMANY
Local recruitment
Recruiting status:
CLOSED
NOVARTIS INVESTIGATIVE SITE
Hatvan
BARANYA, HUNGARY
Local recruitment
Recruiting status:
CLOSED
NOVARTIS INVESTIGATIVE SITE
Secunderabad
TELANGANA, INDIA
Local recruitment
Recruiting status:
CLOSED
NOVARTIS INVESTIGATIVE SITE
Hyderabad
TELANGANA, INDIA
Local recruitment
Recruiting status:
CLOSED
Participating centers
Information source
Last modification :
June 07, 2026
Current data since :
09 Jun 10:18
Data source :
clinicaltrials.gov
