NCT04638309终止1 期
Phase 1 Study to Evaluate Safety and Efficacy of APR-548 in Combination With Azacitidine for the Treatment of TP53-Mutant Myelodysplastic Syndromes
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 4
- 主要终点
- To Investigate the "Number of Participants With Treatment Emergent Adverse Events From Treatment With APR-548 as Monotherapy and in Combination With Azacitidine
研究概览
简要总结
Phase 1 study evaluating the safety and efficacy of APR-548 in combination with Azacitidine for the treatment of TP53-Mutant Myelodysplastic Syndromes.
详细描述
Open-label first-in-human (FIH) phase 1 clinical trial assessing the safety, pharmacokinetics (PK), and clinical activity of orally (p.o.) administered APR-548 alone and in combination with azacitidine for the treatment of TP53-mutant myelodysplastic syndromes (MDS).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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
- •Provision of signed and dated, written informed consent prior to any study specific procedures.
- •Documented diagnosis of TP53-mutant MDS, according to WHO criteria that is relapsed/refractory or previously untreated MDS.
- •Adequate organ function as defined by the following laboratory values:
- •Creatinine clearance ≥60 mL/min (by Cockcroft-Gault method, Appendix I),
- •Total serum bilirubin ≤1.5 × upper limit of normal (ULN) unless due to Gilbert's syndrome or MDS organ involvement,
- •Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5 × ULN, unless due to MDS organ involvement.
- •Age ≥18 years at the time of signing the informed consent form.
- •Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2 (Appendix II).
- •Projected life expectancy of ≥12 weeks.
- •Clear ocular media and adequate pupil dilation to permit fundus examination and retinal imaging.
排除标准
- •Cardiac abnormalities, which includes, but not limited to:
- •Myocardial infarction within six months prior to enrollment
- •New York Heart Association Class III or IV heart failure or known LVEF <40%
- •Concomitant malignancies or previous malignancies with less than a 1 year disease-free interval at the time of signing informed consent.
- •Use of cytotoxic chemotherapeutic agents, or experimental agents for the treatment of MDS within 14 days or 5 half-lives of the product (whichever is shorter) of the first day of study drug treatment.
- •Prior exposure to eprenetapopt (APR-246).
- •A female subject who is pregnant or breast-feeding.
- •Known history of human immunodeficiency virus (HIV), active hepatitis B or active hepatitis C infection.
- •Malabsorption syndrome or other condition likely to affect gastrointestinal absorption of APR-
- •Known history or current evidence of ocular disease in either eye
研究组 & 干预措施
Cohort 1
Experimental
Dose level 1
干预措施: APR-548 + Azacitidine (Drug)
Cohort 2
Experimental
Dose level 2
干预措施: APR-548 + Azacitidine (Drug)
Cohort 3
Experimental
Dose level 3
干预措施: APR-548 + Azacitidine (Drug)
结局指标
主要结局
To Investigate the "Number of Participants With Treatment Emergent Adverse Events From Treatment With APR-548 as Monotherapy and in Combination With Azacitidine
时间窗: Through study completion, approximately 28 days
Occurrence of frequency of treatment emergent adverse events by reviewing safety data including AEs, vital signs, laboratory data, ECG, ophthalmologic assessment findings, and other physical exam findings.
次要结局
未报告次要终点
研究者
研究点 (4)
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