2024-517972-39-00招募中3 期
A phase 3, multicenter, randomized, double-blind, placebo- controlled, parallel-group study with an open-label period and long-term extension to assess the efficacy and safety of rilzabrutinib in participants with warm autoimmune hemolytic anemia (wAIHA)
Sanofi-Aventis Recherche & Developpement39 个研究点 分布在 11 个国家目标入组 58 人开始时间: 2025年7月7日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 58
- 试验地点
- 39
- 主要终点
- Proportion of participants achieving durable Hb response (DHR)
研究概览
简要总结
To evaluate the effect of rilzabrutinib on durable Hb response (DHR) in participants with wAIHA in the PAP
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Male and female participants with a confirmed diagnosis of primary wAIHA for at least 3 months.
- •Participants who have previously failed to maintain a sustained response after treatment with CS (CS-resistance [defined as failure to obtain hemoglobin response within 3 weeks on at least 1 mg/kg or 60 mg prednisone or equivalent per day], CS-dependent wAIHA [defined as need to continue on prednisone or equivalent at a dose of >10 mg/day to maintain a response]), or are intolerant or ineligible to CS (defined as with contraindications, pre-existing medical conditions or CS-related complications that may render CS intolerant or ineligible per the best clinical judgement of the investigators).
- •Participants with Eastern Cooperative Oncology Group (ECOG) performance status Grade 2 or lower.
- •Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
排除标准
- •Participants with clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator.
- •Concurrent treatment with other experimental/investigational drugs within 30 days or 5 half-lives, whichever is longer, prior to treatment start. Participants who previously received treatment with BTK inhibitors for wAIHA before Day 1 (randomization) are not eligible.
- •Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.
- •Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years.
- •Participants with symptomatic herpes zoster within 3 months prior to screening.
- •Participants with secondary wAIHA from any cause including drugs, Evans Syndrome, lymphoproliferative disorders (low count monoclonal B-cell lymphocytosis is allowed), infectious or autoimmune disease, or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed.
- •Participants with history of myelodysplastic syndrome.
- •Participants with uncontrolled or active HBV infection or Active HCV infection.
- •HIV infection.
- •Participants with history of solid organ transplant.
- •Participants with a history of active or latent tuberculosis (TB).
结局指标
主要结局
Proportion of participants achieving durable Hb response (DHR)
Proportion of participants achieving durable Hb response (DHR)
次要结局
- Proportion of participants achieving overall Hb response (Response or Complete Response)
- The time taken (days) to achieve the first Hb increase by ≥2 g/dL from baseline during the PAP in the absence of transfusion and rescue medication
- Change from baseline in fatigue total score as measured by Functional Assessment of Chronic Illness Therapy (FACIT) – fatigue
- Change from baseline in levels of LDH
- Proportion of participants requiring use of rescue therapy after Week 4 of treatment during the PAP
- Change from baseline in dyspnea severity score as measured by FACIT-Dyspnea
- Incidence of treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (SAEs), treatment-emergent adverse events of special interest (AESIs), as well as clinical laboratory evaluations, vital sign, physical exam, and electrocardiograms (ECG)
研究者
Clinical Sciences and Operations
Scientific
Sanofi-Aventis Recherche & Developpement
研究点 (39)
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