Ropustin for Refractory Aplastic Anaemia After Radiotherapy - a Single-centre, Prospective, Open-label, Single-arm Study
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- ORR
研究概览
简要总结
To investigate the efficacy and safety of roprostin in the treatment of refractory AA after radiotherapy.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 years, male or female.
- •Diagnosis consistent with refractory AA after radiotherapy. refractory is defined as patients who have failed to respond to at least an adequate amount of supportive therapy, cyclosporine or povidone TPO-RA for 3 months .
- •At least one of the following conditions was met at enrolment: haemoglobin <90 g/L. Platelets <30 x 10^9/L, neutrophils <1.0 x 10^9/L.
- •Baseline liver and renal function is less than two times the normal value.
- •No active infection.
- •Agreed to sign the consent form.
- •Eastern Cooperative Oncology Group (ECOG) score of 0-2.
排除标准
- •Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).
- •Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).
- •PNH clones ≥50%.
- •Hematopoietic stem cell transplantation (HSCT) prior to enrolment.
- •Prior treatment with ATG.
- •Infection or bleeding uncontrolled by standard therapy.
- •Allergy to roprostin.
- •Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
- •Any concomitant malignancy, localised basal cell carcinoma of the skin within 5 years.
- •Previous history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants.
- •Pregnant or lactating (breastfeeding) women.
- •Participation in another clinical trial within 3 months.
研究组 & 干预措施
Romiplostim group
Enrolled patients were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10^9/L and continuation of roprostin for platelet counts <50 x 10^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 months.
干预措施: Romiplostim (Drug)
结局指标
主要结局
ORR
时间窗: 3 months, 6 months
Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC \< 0.5 x 10\^9/L increased by at least 0.5 x 10\^9/L after treatment, or initial PLT \< 20 x 10\^9/L increased by at least 20 x 10\^9/L after treatment.
CRR
时间窗: 3 months, 6months
Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC \< 0.5 x 10\^9/L increased by at least 0.5 x 10\^9/L after treatment, or initial PLT \< 20 x 10\^9/L increased by at least 20 x 10\^9/L after treatment.
次要结局
- safety events(3 months，6months)