A Study of Bortezomib, Pomalidomide, Dexamethasone in Patients with Systemic AL Amyloidosis
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Hematologic VGPR + CR rate at 6 months
研究概览
简要总结
This is an open-label, multicenter, Phase 2 study in subjects with newly diagnosed or previous treated systemic light chain (AL) amyloidosis. Approximately 40 subjects will receive therapy with bortezomib, pomalidomide, and dexamethasone. The primary outcome is hematologic very good partial response and complete response rate at 6 months.
详细描述
Proteasome inhibitor and immunmodulators have synergistic effect for plasma cell dyscrasia. Due to the fact that more than 70-80% of patients with amyloidosis have renal involvement, the application of lenalidomide is limited. Thus, the investigators designed this open-label, multicenter, phase 2 study for newly diagnosed or previously treated systemic AL amyloidosis with bortezomib, pomalidomide and dexamethasone regimen.
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
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of AL amyloidosis, confirmed by histology and typed with immunohistochemistry, immunoelectron microscopy or mass spectrometry.
- •Newly diagnosed or previous treated AL amyloidosis
- •Patients must be ≥ 18 years of age.
- •ECOG performance status 0, 1 or
- •Measurable disease defined by at least one of the following:
- •① serum free light chain (FLC) ≥2.0 mg/dL (20 mg/L) with an abnormal kappa:lambda ratio or the difference between involved and uninvolved free light chains (dFLC) ≥2mg/dL (20 mg/L).
- •②. presence of a monoclonal spike that is ≥5 g/l.
- •Symptomatic organ involvement (heart, kidney, liver/GI tract, peripheral nervous system).
- •Absolute neutrophil count (ANC) ≥1.0 X 10^9/L, Hemoglobin ≥70 g/L, Platelets ≥50 X 10^9/L
- •eGFR ≥20 mL/min/ 1.73 m^2
- •Written informed consent in accordance with local and institutional guidelines.
- •Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
排除标准
- •Fulfill with the criteria of active multiple myeloma or active lymphoplasmacytic lymphoma.
- •Presence of other tumors which is/are in advanced malignant stage and has/have systemic metastasis;
- •Severe or persistent infection that cannot be effectively controlled;
- •Presence of severe autoimmune diseases or immunodeficiency disease;
- •Patients with active hepatitis B or hepatitis C ([HBVDNA+] or [HCVRNA+]);
- •Patients with HIV infection or syphilis infection;
- •Any situations that the researchers believe will increase the risks for the subject or affect the results of the study.
研究组 & 干预措施
BPd
BPd regimen (Bortezomib, pomalidomide, and dexamethasone) will be applied every 28 days until progression of disease, unacceptable toxicity or subsequent therapy, for a maximum of six cycles.
干预措施: Bortezomib (Drug)
BPd
BPd regimen (Bortezomib, pomalidomide, and dexamethasone) will be applied every 28 days until progression of disease, unacceptable toxicity or subsequent therapy, for a maximum of six cycles.
干预措施: Pomalidomide (Drug)
BPd
BPd regimen (Bortezomib, pomalidomide, and dexamethasone) will be applied every 28 days until progression of disease, unacceptable toxicity or subsequent therapy, for a maximum of six cycles.
干预措施: Dexamethasone (Drug)
结局指标
主要结局
Hematologic VGPR + CR rate at 6 months
时间窗: 6 months
Hematologic very good partial response plus complete response rate at 6 months
次要结局
- TTNT at 2 years(2 years)
- Overall Hematologic response rate at 6 months(6 months)
- At least one organ response at 6 months(6 months)
- TRAE(6 months)
- Estimated PFS at 2 years(2 years)
- Estimated OS at 2 years(2 years)
研究者
Jin Lu, MD
Principle Investigator
Peking University People's Hospital