Warm Autoimmune Hemolytic Anemia
Conditions
Interventions
Arm A (investigational): VAY736 at 3 mg/kg dose, intravenously
Arm B (investigational): VAY736 at 9 mg/kg dose, intravenously
Arm C (control): placebo, intravenously
Sponsors
Yamauchi Kyosuke
Eligibility
Sex/Gender
All
Inclusion criteria
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 >= 5 g/dL and <10 g/dL and presence of symptoms related to anemia at Screening and Week 1 - The dose of supportive care must be stable for at least 4 weeks prior to randomization into the study
Exclusion criteria
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 - No hematologic response to the last course of B-cell depleting therapy - Neutrophils: 1.5 x upper limit of normal (ULN) - 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Binary variable indicating whether a patient achieves a durable response (Hb >=10 g/dL and >=2 g/dL increase from baseline), for a period of at least 8 consecutive weeks, between W9 and W25, in the absence of rescue or prohibited treatment | — |
Countries
Argentine, Australia, China, France, Germany, Hungary, India, Israel, Italy, Japan, Malaysia, Rumania, Singapore, Spain, Taiwan, Thailand, United Kingdom, United States
Contacts
Public ContactKyosuke Yamauchi
Novartis Pharma. K.K.
Outcome results
None listed