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.

JRCT ID: jRCT2031220601

Registered date:01/02/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Warm Autoimmune Hemolytic Anemia
• Date of first enrollment: 12/07/2023
• Target sample size: 12
• Countries of recruitment: Argentine,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 type: Interventional

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 Outcome: 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

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

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

• Primary Sponsor: Yamauchi Kyosuke
• Secondary ID(s): NCT05648968