NCT05423691招募中1 期
Phase Ib, Open-label Study of Add on Therapy With CK0804 in Participants With Myelofibrosis, With Suboptimal Response to Ruxolitinib
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 24
- 试验地点
- 4
- 主要终点
- To determine Treatment limiting toxicity (TLT) as defined below
研究概览
简要总结
To assess the safety and tolerability of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
详细描述
- Safety Run-in
The study will employ a 3+3+3 design to assess the safety and tolerability of the treatment based on treatment-limiting toxicities (TLTs) occurring up to 1 Cycle (28 days) after the first infusion. 2. Expansion
After a total of 9 participants completed 28 days and are evaluated for tolerability in the safety run-in phase, additional participants may be included in the expansion cohort in order to have approximately 24 evaluable myelofibrosis
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
- •Ability to comprehend and willingness to sign a written informed consent form (ICF) for the study.
- •Age above 18 years inclusive at the time of signing the ICF.
- •Participants who fulfill the diagnostic criteria of myelofibrosis including primary myelofibrosis and myelofibrosis arising from polycythemia vera and essential thrombocythemia
- •Life expectancy is greater than 6 months.
- •Subject has been receiving ruxolitinib therapy, is unlikely to benefit from further ruxolitinib monotherapy in the opinion of the investigator; AND meeting the following criteria: receiving ruxolitinib >3 months prior to enrollment; AND stable dose for 8 weeks before starting therapy with CK0804
- •Subject with evidence of evaluable residual burden of disease following ruxolitinib monotherapy treatment, consisting of:
- •presence of grade ≥2 anemia or thrombocytopenia or neutropenia, OR
- •presence of disease-related symptoms, as determined by a Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN SAF TSS) score of ≥10 points, OR
- •documented splenomegaly of at least 5 cm below the costal margin as measured by physical examination or splenomegaly as documented by ultrasound or MRI.
- •Willingness to avoid pregnancy or fathering children based on the criteria below
- •Men must agree to take appropriate precautions to avoid fathering children (with at least 99% certainty) from screening through 90 days after the last study treatment dose and must refrain from donating sperm during this period. Permitted methods that are at least 99% effective in preventing should be communicated to the participants and their understanding confirmed.
- •Women of childbearing potential must have a negative serum pregnancy test at screening before the first dose (within 3 days of the first study treatment dose) and must agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through the safety follow-up visit and must not donate oocytes during this period. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the participants and their understanding confirmed,
- •Women of nonchildbearing potential (ie, surgically sterile with a hysterectomy and/or bilateral oophorectomy OR ≥ 12 months of amenorrhea and at least 50 years of age) are eligible.
- •ECOG performance status of 0 to 2
排除标准
- •Any major surgery within 28 days before the first dose of study treatment.
- •Undergone any prior allogenic or autologous stem cell transplantation or a candidate for such transplantation.
- •Received chemotherapy, immunomodulatory drug therapy, immunosuppressive therapy, biological therapy, endocrine therapy, targeted therapy, antibody or hypomethylating agent to treat the participant's disease, with the exception of ruxolitinib, within 5 half-lives or 28 days (whichever is shorter) before the first dose of study treatment.
- •Participant has received splenic irradiation within the past 6 months.
- •Significant concurrent, uncontrolled medical condition or infections, which in the opinion of the principal investigator may interfere in the study participation.
- •Inability or unlikeliness of the participant to comply with the dose schedule and study evaluations, in the opinion of the investigator.
- •Women who are pregnant or breastfeeding.
- •Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study treatment and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.
- •Participants with laboratory values at screening as defined
- •Platelets < 50 × 10^9/L without the assistance of growth factors, thrombopoietic factors, or platelet transfusions
- •ANC < 0.5 × 10^9/L
- •ALT ≥ 2.5 × ULN
- •AST ≥ 2.5 × ULN
- •Direct Bilirubin > 2.0 × ULN
- •ALP ≥ 3 × ULN
- •Creatinine clearance < 50 mL/min according to Cockcroft-Gault formula.
- •Unwillingness to be transfused with blood components including RBC and platelet transfusions.
- •Inability of the participant (or parent, guardian, or legally authorized representative) to comprehend the ICF or unwillingness to sign the ICF.
研究组 & 干预措施
Arm 1
Experimental
CK0804 will be administered intravenously (IV) 100 million Treg Cells every 28 days up to 6 infusions.
干预措施: CK0804 (Drug)
结局指标
主要结局
To determine Treatment limiting toxicity (TLT) as defined below
时间窗: 28 days
* severe (grade 3 or 4) infusion-related toxicity within 24 hours (NCI-CTCAE V5.0) of exposure that does not resolve with standard of care treatment within 72 hours. * regimen related death within 28 days
次要结局
- Assessment of overall response rate (ORR) (measured as CR or PR) and its duration, using modified International Working Group-Myeloproliferative Neoplasm Research and Treatment (IWG-MRT) and European Leukemia Net (ELN) consensus report.(6 months)
- Rate of anemia response as per modified IWG-MRT ELN response criteria.(6 months)
- Rate of spleen response by imaging at and after 24 weeks as per IWG-MRT ELN response criteria(6 months)
- Percentage of Participants who will Achieve Total Symptom Score Reduction Greater Than or Equal to (≥) 50% (TSS50) as Measured by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)(6 months)
研究者
研究点 (4)
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