NIPH Clinical Trials Search

JAPANESE
国立保健医療科学院
JRCT ID: jRCT2031220601

Registered date:01/02/2023

A phase 3, randomized, double-blind, study of ianalumab (VAY736) versus placebo in warm autoimmune hemolytic anemia (wAIHA) patients who failed at least one line of treatment.

Basic Information

Recruitment status Recruiting
Health condition(s) or Problem(s) studiedWarm Autoimmune Hemolytic Anemia
Date of first enrollment12/07/2023
Target sample size12
Countries of recruitmentArgentine,Japan,Australia,Japan,China,Japan,France,Japan,Germany,Japan,Hungary,Japan,India,Japan,Israel,Japan,Italy,Japan,Malaysia,Japan,Rumania,Japan,Singapore,Japan,Spain,Japan,Taiwan,Japan,Thailand,Japan,United Kingdom,Japan,United States,Japan
Study typeInterventional
Intervention(s)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

Outcome(s)

Primary OutcomeBinary 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
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum>= 18age old
Age maximumNot applicable
GenderBoth
Include 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
Exclude 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: <1000/mm3 - Serum creatinine >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

Related Information

Contact

Public contact
Name Kyosuke Yamauchi
Address Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan Tokyo Japan 105-6333
Telephone +81-120-003-293
E-mail rinshoshiken.toroku2@novartis.com
Affiliation Novartis Pharma. K.K.
Scientific contact
Name Kyosuke Yamauchi
Address Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan Tokyo Japan 105-6333
Telephone +81-120-003-293
E-mail rinshoshiken.toroku2@novartis.com
Affiliation Novartis Pharma. K.K.