Daratumumab and Dexamethasone Combined With Pomalidomide (DPD) or ASCT in the Treatment of Newly Diagnosed Systemic Light Chain Amyloidosis (AL Amyloidosis): a Prospective, Single Center Clinical Trial
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Organ response rate
研究概览
简要总结
This is a prospective, single-center study exploratory clinical trial, aim to exploring the efficacy and safety of daratumumab in patients with AL amyloidosis, patients were divided into three groups: one group received long-term treatment with daratumumab based regimen, and the other group received autologous stem cell transplantation after two standard treatment courses with daratumumab based regimen, and the third group consists of newly diagnosed stage IIIb AL amyloidosis patients who plan to receive DPD treatment. The purpose of this study is to observe the efficacy and safety of Daratumumab, in the treatment of newly diagnosed systemic AL amyloidosis.
详细描述
This is a prospective, single-center study exploratory clinical trial, aim to exploring the efficacy and safety of daratumumab in patients with AL amyloidosis, patients were divided into three groups: one group received long-term treatment with daratumumab based regimen (group A) and the other group received autologous stem cell transplantation after two standard treatment courses with daratumumab based regimen (group B) , and the third group consists of newly diagnosed stage IIIb AL amyloidosis patients who plan to receive DPD treatment (group C). Participants with AL Amyloidosis will receive the drug daratumumab by IV infusion once weekly for two months, then every 2 weeks for four months, then once each month. Study treatment may continue until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression. Group C were given Daratumumab (16mg/kg), dexamethasone (40mg) and pomalidomide. The usage of Daratumumab and dexamethasone is the same as group A and B. Patients received oral pomalidomide on days 1-21 of a 28-days cycle from cycle 1 to 6. The dose of pomalidomide is adjusted based on the patient's renal function and ECOG score.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must be aged ≥18 and ≤75 years;
- •Newly diagnosed AL amyloidosis ((confirmed by pathological examination with at least one major organ involvement (heart, kidney, or liver));
- •In group A and B, according to the Mayo 2004 staging system, the disease is classified as stage Ⅰ-ⅢA; In group C, according to the Mayo 2004 staging system, the disease is classified as stage IIIB: NT⁃proBNP>8500ng/L and cTnT>0.035μg/L or cTnI>0.01g/L;
- •Participants must personally sign an informed consent form approved by the Ethics Committee before the start of the study;
- •Expected survival ≥ 12 weeks;
- •ECOG performance status≤ 2;
- •Female participants of childbearing potential must agree to use effective contraception from the day of signing the informed consent until 365 days after the infusion. Effective contraception is defined as abstinence or the use of a contraceptive method with a failure rate of <1% per year.
排除标准
- •eGFR< 30ml/min/1.73m2;
- •Combined multiple myeloma;
- •Acute or chronic infection requiring treatment within 30 days prior to baseline;
- •Pregnant or breastfeeding women.
- •Participants known to have life-threatening allergic reactions, hypersensitivity, or intolerance to Monoclonal antibodies or immune modulators.
- •Other conditions deemed by the researcher as unsuitable for enrollment.
研究组 & 干预措施
long-term daratumumab treatment group (group A)
The patient will receive the standard protocol of daratumumab combined with dexamethasone treatment, once a week for the first two months, once every two weeks for four months, and then once a month for 18 months.
干预措施: Dratumumab / Hyaluronidase Injection [Darzalex Faspro] (Drug)
daratumumab combine with ASCT treatment group (group B)
This group of patients will first receive two courses of treatment with the standard protocol of daratumumab combined with dexamethasone. Subsequent patients will receive ASCT treatment, and patients who achieve VGPR or CR after transplantation treatment will be followed up for observation
干预措施: Dratumumab / Hyaluronidase Injection [Darzalex Faspro] (Drug)
daratumumab combine with ASCT treatment group (group B)
This group of patients will first receive two courses of treatment with the standard protocol of daratumumab combined with dexamethasone. Subsequent patients will receive ASCT treatment, and patients who achieve VGPR or CR after transplantation treatment will be followed up for observation
干预措施: autologous stem cell transplantation (ASCT) (Procedure)
Daratumumab and Dexamethasone combined with pomalidomide (DPD) (group C)
newly diagnosed stage IIIb AL amyloidosis patients will received Daratumumab (16mg/kg), dexamethasone (40mg) and pomalidomide. The usage of Daratumumab and dexamethasone is the same as group A. Patients received oral pomalidomide on days 1-21 of a 28-days cycle from cycle 1 to 6. The dose of pomalidomide is adjusted based on the patient's renal function and ECOG score.
干预措施: Dratumumab / Hyaluronidase Injection [Darzalex Faspro] (Drug)
Daratumumab and Dexamethasone combined with pomalidomide (DPD) (group C)
newly diagnosed stage IIIb AL amyloidosis patients will received Daratumumab (16mg/kg), dexamethasone (40mg) and pomalidomide. The usage of Daratumumab and dexamethasone is the same as group A. Patients received oral pomalidomide on days 1-21 of a 28-days cycle from cycle 1 to 6. The dose of pomalidomide is adjusted based on the patient's renal function and ECOG score.
干预措施: Pomalidomide 4 MG (Drug)
结局指标
主要结局
Organ response rate
时间窗: 1 year
the 1-year organ response rate after treatment
Hematological complete response rate
时间窗: 6 months
the 6 months overall hematologic complete response rate after treatment.
次要结局
- Time to Next Treatment(3 years)
- Overall survival(5 years)
- Progression-free survival(3 years)
研究者
Zhi-Hong Liu, MD
Professor
Nanjing University School of Medicine