A Phase 1b, Open-label, Study of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance and Other Measures of Pharmacodynamic Activity
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 12
- 试验地点
- 3
- 主要终点
- To Determine the impact of multiple doses of RCT1100 on MCC
研究概览
简要总结
This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
详细描述
The primary objective of this study is to determine the impact of multiple doses of inhaled RCT1100, administered via nebulizer, on MCC with adult participants with Primary Ciliary Dyskinesia caused by pathogenic mutations in the DNAI1 Gene.
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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant is a male or female, 18 to 75 years of age, inclusive, at the time of consent.
- •Participant has disease-causing (pathogenic and/or likely pathogenic) mutations in the DNAI1 gene.
- •The participant has a percent predicted forced expiratory volume in 1 second (FEV1pp) of at least 40% predicted.
排除标准
- •History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.
- •History of cancer, with exception of adequately treated basal cell or squamous cell carcinoma of the skin.
- •Predisposition to bleeding or clinically meaningful hemorrhagic event in the 12 months prior
- •Medically significant hemoptysis.
- •Anticoagulation therapy for the treatment of a pulmonary embolus or has had a pulmonary embolus in the last 6 months of screening.
- •Active tuberculosis infection.
- •12-lead ECG with QT interval >450 msec (or >480 msec for BBB)
- •Laboratory abnormalities in clinical laboratory tests at screening:
- •Serum creatinine level
- •Total bilirubin, aspartate aminotransferase or alanine aminotransferase values
- •Hematological or coagulation values outside the normal reference range
- •Any medical history of disease that has the potential to cause a rise in total bilirubin over the ULN.
- •COVID-19 infection within 4 weeks of Screening or receipt of COVID-19 vaccine within 2 weeks prior to first dose of RCT
- •Receipt of vaccine with live virus, attenuated live virus, or live viral components within 2 weeks prior to first dose of RCT1100 or to receive these vaccines during treatment or within 8 weeks of completion of study treatment.
- •Other protocol defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
PCD Participants
RCT1100 mRNA therapy supplied to eligible participants with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene
干预措施: RCT1100 (Drug)
结局指标
主要结局
To Determine the impact of multiple doses of RCT1100 on MCC
时间窗: Baseline through Week 12
The Change in Mucociliary and Cough Clearance through 1 hour after radiotracer administration by either the albumin-based PRMC or sulfur-colloid technique
次要结局
未报告次要终点