NCT06616415已完成4 期
A Multicenter, Open-label, Single-arm, Phase 4 Clinical Study to Evaluate the Pharmacokinetics, Efficacy, and Safety of Belumosudil Mesylate Tablets in Chinese Adolescents (Aged From 12 to Less Than 18 Years) With Chronic Graft-versus-host Disease (cGVHD) Who Have Had an Inadequate Response to Glucocorticoids or Other Systemic Therapies
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 6
- 试验地点
- 4
- 主要终点
- Plasma concentrations of belumosudil at specified time points
研究概览
简要总结
This is a single group, Phase 4, single-arm post-marketing study for treatment.
The purpose of this study is to verify the pharmacokinetics, efficacy, and safety of belumosudil mesylate tablets in Chinese adolescent participants (aged from 12 to less than 18) with cGVHD who have had an inadequate response to glucocorticoids or other systemic therapies.
Participants will receive treatment with belumosudil tablets 200 mg once daily in 28-day cycles during the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be 12 to less than 18 years of age at the time of signing the informed consent.
- •Participant has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
- •Has active moderate to severe cGVHD.
- •Has received at least one line of prior systemic therapy for cGVHD.
- •Participant must receive a corticosteroid therapy for cGVHD with a stable dose for at least 2 weeks prior to the first dose of the IMP.
- •Has a Lansky-Play performance score of ≥
- •Participants should have an expected survival of longer than 6 months.
- •Body weight of 30 kg and above.
- •Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •The participant or their legally authorized representative (LAR) must be capable of giving signed informed consent.
排除标准
- •Participants are excluded from the study if any of the following criteria apply:
- •Recurrence of hematologic neoplasms (according to the corresponding criteria for recurrence of primary hematologic neoplasms) or post-transplant lymphoproliferative disease at screening.
- •Received investigational systemic therapy for cGVHD within 28 days prior to enrollment, unless the prior treatment had been washed out for at least 28 days or 5 half-lives prior to enrollment, whichever is shorter.
- •Absolute neutrophil count (ANC) <1.0 × 10^9/L.
- •Platelet count <50 × 10^9/L.
- •Alanine aminotransferase (ALT) >3× the upper limit of normal (ULN), aspartate aminotransferase (AST) >3 × ULN.
- •Total bilirubin (TBIL) >1.5 × ULN (>3 ULN if Gilbert's syndrome).
- •Estimated Glomerular Filtration Rate (eGFR) <30 mL/min/1.73 m^2 using the revised Bedside Schwartz formula . Revised Schwartz equation: CrCl (mL/min/1.73 m^2) = 0.413 × (height [in cm])/Creatinine (in mg/dL) at screening visit.
- •Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
研究组 & 干预措施
Belumosudil
Experimental
Participants will receive belumosudil 200 mg tablets orally QD in 28-day cycles until clinically significant progression of cGVHD
干预措施: Belumosudil (Drug)
结局指标
主要结局
Plasma concentrations of belumosudil at specified time points
时间窗: At Day 1 and Day 29(±3)
次要结局
- Change in symptom activity assessed based on patient-reported cGVHD activity(Up to 18 month)
- System organ response rate(Up to 18 month)
- Overall response rate (ORR)(Up to 18 month)
- Duration of response (DoR)(Up to 18 month)
- Number and proportion of participants with dose reduction in corticosteroid during the treatment period(Up to 18 month)
- Failure-free survival (FFS)(Up to 18 month)
- Overall survival (OS)(Up to 18 month)
- Change in cGVHD severity based on physician-reported overall cGVHD activity(Up to 18 month)
- Modified Lee cGVHD Symptom Scale change(Up to 18 month)
- Number and proportion of participants with calcineurin inhibitor (CNI) dose reduction or discontinuation during the treatment period(Up to 18 month)
- Number of participants with treatment-emergent adverse events (TEAEs), serious TEAEs, and adverse events of special interest (AESIs)(Up to 18 month)
研究者
研究点 (4)
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