A Phase 1b Randomized, Double-blind, Placebo-Controlled Trial to Evaluate the Safety of CB-280 in Patients With Cystic Fibrosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 32
- 试验地点
- 20
- 主要终点
- Determine the safety and tolerability of CB-280 in adult cystic fibrosis patients: incidence and severity of adverse event (AEs) assessed by Common Terminology Criteria for Adverse Events, version 5 (CTCAE v5.0)
研究概览
简要总结
This is a phase 1b multiple ascending dose escalation study to evaluate the safety and tolerability of arginase inhibitor CB-280 in subjects with cystic fibrosis.
详细描述
Study CX-280-202 is a Phase 1b, randomized, double-blind, placebo-controlled, multiple ascending dose escalation study of CB-280 in adult subjects with cystic fibrosis and chronic infection with Pseudomonas aeruginosa. The study will evaluate the safety, pharmacokinetics, pharmacodynamics, and biological activity of CB-280 in approximately 32 adult patients with cystic fibrosis. There are four planned sequential dose escalation cohorts of 8 subjects each, randomized 6:2 to receive CB-280 or matched placebo at doses of 50 mg, 100 mg, 200 mg, or 400 mg administered twice daily for 14 days. Intermediate dose levels may be evaluated based on emerging safety data at the planned dose levels.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
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
- •Written Informed Consent in accordance with federal, local, and institutional guidelines
- •Confirmed diagnosis of cystic fibrosis
- •Male or female subjects ≥ 18 years on the date of informed consent
- •Percent predicted FEV1 of 40-90% at screening per Global Lung Function Initiative (GLI) equation
- •Clinically stable with no significant changes in health status within 28 days prior to Day 1
- •Chronic lung infection with P. aeruginosa defined as at least one positive culture in the last two years and more than 50% of cultures positive since then
- •Stable cystic fibrosis medication regimen for at least 28 days inclusive of CFTR modulators prior to Day 1
- •Hemoglobin > 10 g/dL at screening
- •Glomerular filtration rate > 50 mL/min/1.73 m2 at screening
- •Normal liver function at screening
排除标准
- •History of any comorbidity that, in the opinion of the Investigator, might pose an additional risk in administering study drug to the subject or confound the results of the study
- •Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus)
- •Unable to receive study medication per os (PO)
- •Females who are pregnant, have a positive pregnancy test at screening, or are nursing (lactating)
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Cohort 1
CB-280 twice daily at 50 mg for 14 days
干预措施: CB-280 (Drug)
Cohort 2
CB-280 twice daily at 100 mg for 14 days
干预措施: CB-280 (Drug)
Cohort 3
CB-280 twice daily at 200 mg for 14 days
干预措施: CB-280 (Drug)
Cohort 4
CB-280 twice daily at 400 mg for 14 days
干预措施: CB-280 (Drug)
Placebo
Placebo twice daily for 14 days
干预措施: Placebos (Drug)
结局指标
主要结局
Determine the safety and tolerability of CB-280 in adult cystic fibrosis patients: incidence and severity of adverse event (AEs) assessed by Common Terminology Criteria for Adverse Events, version 5 (CTCAE v5.0)
时间窗: Start of treatment to Day 28
次要结局
- Pharmacokinetics of plasma CB-280 measured by Peak Plasma Concentration (Cmax)(Day 14)
- Pharmacokinetics of plasma CB-280 measured by area under the plasma concentration versus time curve, from time 0 to the last observed non-zero concentration (AUC 0-t)(Day 14)