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

• Register: ClinicalTrials.gov
• Last refreshed on: 30 June 2025
• Main ID: NCT05648968
• Date of registration: 23/11/2022
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia VAYHIA
• Scientific title: 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
• Date of first enrolment: December 30, 2022
• Target sample size: 90
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT05648968
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, China, France, Germany, Hungary, India, Israel, Italy, Japan, Malaysia, Romania, Singapore, Spain, Taiwan, Thailand, United Kingdom, United States

Contacts

• Name: Novartis Pharmaceuticals

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion 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.


Age minimum: 18 Years
Age maximum: 100 Years
Gender: All

Health Condition(s) or Problem(s) studied

Warm Autoimmune Hemolytic Anemia (wAIHA)

Intervention(s)

Biological: Ianalumab

Drug: Placebo

Primary Outcome(s)

Binary variable indicating whether a patient achieves a durable response [Time Frame: Randomization to Week 25]

Secondary Outcome(s)

Change from baseline in the T-score of PROMIS Fatigue-13a questionnaire [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Ianalumab PK parameter - AUClast [Time Frame: After first dose of study treatment (pre-dose, 2 hours, 336 hours, 672 hours post dose) and after last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).]

Ianalumab PK parameter - Cmax [Time Frame: After first dose of study treatment (pre-dose, 2 hours, 336 hours, 672 hours post dose) and after last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).]

Time from randomization to start of first response in each treatment group [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Time to first occurrence of B cell recovery, defined as =80% of baseline or =50 cells/µL [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Complete response rate [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Change from baseline in immunoglobulin levels [Time Frame: Randomization until month 30]

Time from randomization to start of durable response in each treatment group [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Ianalumab PK parameter - AUCtau [Time Frame: After first dose of study treatment (pre-dose, 2 hours, 336 hours, 672 hours post dose) and after last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).]

Number of participants who received rescue treatment (overall & by type of rescue treatment) [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Percentage of participants who received rescue treatment (overall & by type of rescue treatment) [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Change from baseline in the the frequency and absolute number of CD19+ B cell counts [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Duration of response (Key Secondary) [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Change from baseline in the 8 domain scores and in the summary scores (PCS, MCS) of SF-36 questionnaire [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Ianalumab PK parameter - Tmax [Time Frame: After first dose of study treatment (pre-dose, 2 hours, 336 hours, 672 hours post dose) and after last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).]

Time from randomization to start of complete response in each treatment group [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Hemoglobine Levels [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Immunogenicity of ianalumab [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Ianalumab PK parameter - Accumulation ratio Racc [Time Frame: After last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).]

Response rate [Time Frame: Randomization to end of study (up to 39 months after randomization of last patient)]

Secondary ID(s)

CVAY736O12301

2022-001773-31

2024-510635-21-00

Source(s) of Monetary Support

Please refer to primary and secondary sponsors