2024-510635-21-00招募中3 期
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 (VAYHIA)
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 32
- 试验地点
- 24
- 主要终点
- 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 weeks, between W9 and W25, in the absence of rescue or prohibited treatment
研究概览
简要总结
To demonstrate that either dose of ianalumab induces durable hemoglobin (Hb) response compared to placebo in patients with wAIHA. The primary clinical question of interest is: What is the effect of either dose of ianalumab versus placebo (with or without supportive care), in inducing durable hemoglobin response, in wAIHA patients ≥18 years of age who failed at least one line of treatment, regardless of premature discontinuation from study treatment or temporary treatment interruption/delay, and in the absence of rescue or prohibited treatment?
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Written informed consent form (ICF) must be obtained prior to any screening assessments
- •Male or female participants aged 18 years and older on the day of signing the ICF
- •Participants with primary or secondary wAIHA (previously documented by positive direct antiglobulin test (DAT) 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 corticosteroid resistance, dependence, or intolerance
- •Hemoglobin concentration ≥5 g/dL and <10 g/dL and presence of symptoms related to anemia at Screening and Week
- •The dose of supportive care must be stable for at least 4 weeks prior randomization.
排除标准
- •Patients with wAIHA secondary to hematologic disease involving bone marrow (e.g., chronic lymphocytic leukemia (CLL)) or another disease requiring prohibited medication. Of note, the patients with autoimmune diseases like lupus nephritis (LN), systemic lupus erythematosus (SLE), Primary Sjögren’s Syndrome (pSS) or autoimmune hepatitis (AIH) after wash-out from the treatments are allowed
- •Prior use of B-cell depleting therapy: • within 12 weeks prior randomization, or • no hematologic response to the last course of B-cell depleting therapy, irrespective of time of administration
- •Active viral, bacterial, or other infections (including active or latent tuberculosis or SARSCoV- 2) requiring systemic treatment at the time of screening or history of recurrent clinically significant infection
- •Known history of primary or secondary immunodeficiency, or patients that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAb)-positive. Refer to Section 5.2 exclusion criterion #7b for exemptions applicable for patients who are HBsAg negative and HBcAb positive.
- •Live or live-attenuated vaccination within 4 weeks before randomization
研究组 & 干预措施
-
Auxiliary
Participants receiving -
干预措施: - (Drug)
结局指标
主要结局
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 weeks, between W9 and W25, in the absence of rescue or prohibited treatment
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 weeks, between W9 and W25, in the absence of rescue or prohibited treatment
次要结局
- Duration of response
- Time from randomization to achievement of durable response, to first response, to first complete response
- Response rate, complete response rate and hemoglobin level
- Number and proportion of participants that receive rescue treatment overall and by type of rescue treatment
- Time-standardized numbers of each type of rescue treatment
- Change from baseline in time-standardized number of transfusions
- Frequency of AEs and other safety parameters
- Ianalumab concentration in serum and PK parameters after the first and last dose
- B-cell levels: • Change from baseline in the frequency and absolute number of CD19+ B-cell counts • Time to first occurrence of B-cell recovery, defined as ≥80% of baseline or ≥50 cells/μL
- Immunoglobulins: • Change from baseline in immunoglobulin levels
- Incidence and titer of anti-drug antibodies (ADA) in serum over time
- Change from baseline in dedicated Patient Reported Outcomes (PROs)
研究者
Novartis Pharma Arzneimittel GmbH
Scientific
Novartis Pharma AG
研究点 (24)
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