NCT04994002撤回1 期
A Phase 1b, Open-Label, Pharmacokinetic, Pharmacodynamic, Safety and Tolerability Study of CERC-006 in Adults (Aged 18-31 Years) With Complex Lymphatic Malformations
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 试验地点
- 3
- 主要终点
- Safety and Tolerability of CERC-006 in Adults (Aged 18-31 Years) with Active, Moderate to Severe Complex Lymphatic Malformations
研究概览
简要总结
The primary objective of this study is to evaluate the safety and tolerability of CERC-006 in adults (aged 18-31 years) with active, moderate to severe complex lymphatic malformations.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 31 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must fulfill the following requirements to be eligible for the study:
- •Participant is 18 to 31 years of age (inclusive) at the time of consent.
- •Participant has a verified diagnosis of a complex lymphatic malformation. Other moderate to severe vascular anomalies with associated lymphatic involvement will be considered, with approval by the study medical monitor.
- •Participant's complex lymphatic malformation is considered, in the opinion of the investigator, to be moderate to severe.
- •Participant has adequate liver function defined as:
- •Total bilirubin (sum of conjugated and unconjugated) ≤1.5 × upper limit of normal (ULN)
- •Aspartate transaminase/Alanine aminotransferase (AST/ALT) <5 × ULN
- •Serum albumin > 2 g/dL
- •Participant has fasting low-density lipoprotein (LDL) of <160 mg/dL.
- •Participant has adequate bone marrow function defined as:
- •Peripheral absolute neutrophil count (ANC) > 1000/µL
- •Hemoglobin > 8.0 g/dL
- •Platelet count ≥ 50,000/µL
- •Participant has adequate renal function defined as:
- •Serum creatinine ≤ 1.5 mg/dL or calculated creatinine clearance of > 50 mL/min according to the Cockcroft-Gault equation
- •Participant has agreed to and met the washout period as follows:
- •At least 14 days prior to initiation of CERC-006 if receiving sirolimus (also known as rapamycin), mitogen-activated protein kinase (MEK) inhibitors, phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA) inhibitors, interferon alfa 2b, vascular endothelial growth factor receptor 3 (VEGFR-3) inhibitors, and/or other systemic agents targeting lymphatic malformation
- •At least 7 days prior to initiation of CERC-006 if receiving topical agents targeting lymphatic malformation
- •Participant has a Karnofsky performance status of ≥50%.
排除标准
- •The presence of any of the following criteria excludes a participant from the study:
- •Participant has a concurrent severe or uncontrolled medical disorder, which could compromise participation in the study.
- •Participant has significant impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of CERC-
- •Participant has taken any medication that is a strong cytochrome P450 3A4 (CYP3A4) enzyme inducer or inhibitor within 2 weeks prior to first dose of study drug, or in the opinion of the Investigator, subject may require such medication during the study
- •Participant is receiving chronic treatment with systemic steroids or another immunosuppressive agent, or in the opinion of the Investigator, subject may require such medication during the study
- •Participant has undergone myelosuppressive chemotherapy within 2 weeks, or radiation within 4 weeks prior to first dose of study drug.
- •Participant has a known history of uncontrolled hypertension, cardiovascular disease, advanced arteriosclerosis, structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, heart failure, exercise-related cardiac events including syncope and pre-syncope, or a known family history of sudden cardiac death or ventricular arrhythmia.
- •Participant has received treatment with a medication that has the potential to prolong the QT interval within 1 week prior to the first dose of study drug, or in the opinion of the Investigator, subject may require such medication during the study.
研究组 & 干预措施
Cohort 1: CERC-006 (0.5 mg)
Experimental
Approximately 5 participants will receive CERC-006 at a dose of 0.5 mg twice daily for 28 days.
干预措施: CERC-006 (Drug)
Cohort 2: CERC-006 (1 mg)
Experimental
Following a safety review, if there are no clinically important safety findings in Cohort 1, a second cohort of approximately 5 participants will be enrolled to receive CERC-006 at a dose of 1 mg twice daily for 28 days.
干预措施: CERC-006 (Drug)
结局指标
主要结局
Safety and Tolerability of CERC-006 in Adults (Aged 18-31 Years) with Active, Moderate to Severe Complex Lymphatic Malformations
时间窗: Up to approximately 6 weeks
Safety and tolerability will be assessed by incidence of adverse events (AEs). Clinically significant changes from baseline in vital signs, ocular exams, cardiology tests including echocardiogram (ECHO) and electrocardiogram (ECG), and clinical laboratory results will be recorded as AEs.
次要结局
- Serum Concentrations of CERC-006(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
- Change from Baseline in Karnofsky Performance Status Score(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
- Change from Baseline in Pain Scale Assessment(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
- Change from Baseline in Levels of mTOR Related Pathway Biomarkers(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
- Change from Baseline in 36-Item Short Form Health Survey (SF-36) Score(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
- Change from baseline to end of treatment in radiologic assessment, if clinically indicated and/or clinical signs/symptoms of disease(Baseline (Screening) up to 3 days after last dose of study drug (up to approximately 8 weeks))
研究者
研究点 (3)
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