NCT05102370已完成1 期
A Pilot Study of Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 23
- 主要终点
- Best Response
研究概览
简要总结
Study researchers think that a drug called enasidenib may help people with clonal cytopenia of undetermined significance (CCUS) because the drug blocks the mutated IDH2 protein, which may improve blood cell counts. The purpose of this study is to find out whether enasidenib is a safe and effective treatment for CCUS.
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 at the time of signing the informed consent form.
- •Willing and able to adhere to the study visit schedule and other protocol requirements.
- •Unexplained cytopenia for at least 6 months. Cytopenia is defined as the presence of ≥1 blood count indexes below the following thresholds:
- •Hgb <10 g/dL
- •ANC <1.8 × 10^9/L
- •Platelets <100 × 10^9/L
- •Results of bone marrow biopsy within 1 month of study entry (screening bone marrow biopsy) must not indicate hematologic disease.
- •IDH2 gene mutation (R140 or R172), performed locally, at a frequency >2%.
- •ECOG performance status of 0-
- •Adequate organ function, defined as:
- •Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤3x upper limit of normal (ULN), unless the subject has Gilbert's syndrome.
- •Serum total bilirubin <1.5x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis. In these cases, approval from the study Principal Investigator is required.
- •Creatinine clearance greater than 50 mL/min based on the Cockroft-Gault glomerular filtration rate estimation.
- •Patients being enrolled on study on the basis of anemia, will only be eligible if folate, B12, serum iron, serum ferritin, total iron binding capacity, haptoglobin and peripheral smear within normal limits
- •Women of childbearing potential may participate provided they have a negative serum pregnancy test at screening and a negative serum or urine pregnancy test within 72 h of starting treatment.
- •Women of childbearing potential (WOCBP) and males with partners who are WOCBP must agree to abstain from sexual intercourse or to use 1 highly effective form of contraception during the study and for at least 4 months following the last dose of enasidenib. Males with partners who are WOCBP must agree to use a barrier method.
排除标准
- •Active malignancy defined as >1-cm disease on most recent CT scan in the past 6 months or recent history of cancer (i.e. within the past 5 years) with >50% chance of cancer recurrence in the next 5 years.
- •Current or prior history of hematologic malignancy.
- •Therapy (including maintenance therapy) for solid-tumor malignancy within the last 6 months.
- •Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally.
- •Active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment).
- •Positive direct Coombs test
- •Evidence of hypersplenism on physical exam
- •Pregnant or lactating (women).
研究组 & 干预措施
Participants with CCUS with mutations in IDH2
Experimental
Participants will have CCUS with mutations in IDH2
干预措施: Enasidenib (Drug)
结局指标
主要结局
Best Response
时间窗: Up to 18 months
The primary objective is to estimate the rate of hematologic improvement. The endpoint is evaluated as the best response at any point in up to 18 months of treatment with enasidenib.
次要结局
未报告次要终点
研究者
研究点 (23)
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