A Phase I Single-arm, Open-label Prospective Study to Evaluate the Efficacy and Safety of Peripheral Blood Derived Autologous Multi-lineage Potential Cells (AMPC) in Relapsed/Refractory Acute Myeloid Leukemia (AMPCAL Study)
试验速览
- 阶段
- 1 期
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Overall response rate (ORR) of AMPC in refractory/relapsed AML at 12 months
研究概览
简要总结
A private trial for evaluating the overall response rate contributed by AMPC in AML in refractory or relapsed AML
详细描述
After inclusion and exclusion criteria has been determined and approved, written informed consent will be obtained from the candidate. All medical history relevant to the diagnosis of AML will be collected.
Screening period:
The screening period could extend from 0 to 7 days depending on the completion of screening laboratory results as below.
On day -5 (up to day -1), the patient will undergo a screening test for the following test items:
- BUN, creatinine, electrolyte, liver function test (LFT)
- Full blood count (FBC), including blood smear
- Hepatitis B/C
- Human T lymphocytic virus type I and II (HTLV-I/II)
- HIV1/2
- Syphilis serology
- Mycoplasma serology For Hepatitis B/C,HTLV-I/II, HIV1/2, Syphilis serology, and Mycoplasma serology, these tests obtained up to 3 months prior to day -5 can be allowed for using as screening result.
- Chest X-ray
- Bone marrow study including aspiration with wright's stain, biopsy, flow cytometry, and chromosome study (Any molecular testing for AML is optional.).
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
- 20 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must be unequivocally diagnosed with AML according to WHO classification with accompanying bone marrow biopsy and blood panel results
- •Must have refractory AML, defined as disease unresponsive to initial treatment; or relapsed AML that re-occured after treatment with conventional high dose chemotherapy
- •Candidates who have no available match-sibling donor for bone marrow transplantation (BMT) or are not suitable for BMT due to any reason.
- •Must have had prior treatment with chemotherapy at least 30 days prior to day 0 of this study and have recovered from treatment-related toxicity of chemotherapeutic agents with the exception of persistent diseases
- •Age 20 to 60 years old
排除标准
- •Candidates who received any investigational therapies 4 weeks prior to treatment with this protocol
- •Candidates who received radiotherapy within 4 weeks prior to the treatment of this protocol
- •Candidates who have not recovered from any AE caused by radiotherapy or any agents received 4 weeks earlier
- •Candidates who have had a prior allogeneic stem cell transplant
- •Known case of extramedullary myeloid tumor (myeloid sarcoma)
- •Pregnant or breastfeeding women
- •Hydroxyurea has been prescribed within 10 days prior to day-5
- •Candidates have any abnormal screening laboratory results as below;
- •Hemoglobin < 9 g/dL
- •Total white blood cells count > 30,000/microL (without ongoing G-CSF therapy)
- •Platelet count < 75,000/microL
- •Creatinine clearance < 30 mL/min/1.73 m2 (by Cockcroft and Gault formula)
- •ALT > 5x upper normal limit
- •Bone marrow study at screening period show blast > 40% of total nucleated cells or severe hypocellularity (defined as < 25% of normal cellularity for corresponding age) with presence of cluster of blasts
- •Candidates have active heart disease including recent or chronic heart failure, unstable angina, recent acute myocardial infarction, or significant arrhythmia within 6 months of recruitment.
- •Candidates have concurrent malignancies unless the candidates has been free of the disease for at least 5 years.
- •Candidates positive for HIV1/2, hepatitis B/C, HTLVI/II, and Syphilis
结局指标
主要结局
Overall response rate (ORR) of AMPC in refractory/relapsed AML at 12 months
时间窗: 12 months
Overall response rate (ORR) is defined as whether the patient achieves complete remission (CR) or complete remission with incomplete blood count recovery (CRi) CR Requirements: * Bone marrow aspiration shows less than 5% of abnormal blasts as determined by evidence from flow cytometry or immunohistochemistry * Bone marrow biopsy shows no clusters of blast cell * Normal values for absolute neutrophil count in peripheral blood exceeds 1,000/microL * Platelet count in peripheral blood exceeds 100,000/microL * Absence of extramedullary AML CRi Requirements: * All parameters of CR except platelet recovery or neutrophil recovery * Incomplete recovery-platelet count is less than 100,000/microL or neutrophil count less than 1,000/microL in peripheral blood
次要结局
- Overall survival (OS) rate at 12 months(12 months)
- ORR of AMPC in refractory/relapsed AML at 3 and 6 months(3 and 6 months)
- Safety profile and treatment-related adverse events (AE) upto 12-month follow up period(12 months)
- Time-to-next treatment (TTNT), defined as the time from the start of AMPC therapy to the start date of a subsequent line of therapy.(12 months)
研究者
Supachai Ekwattanakit
Principle Investigator
Lai Corporation Pty. Ltd.