A Phase 1b, Single-Center, Open-Label Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 7
- 试验地点
- 2
- 主要终点
- The number of participants with Adverse Events (AEs), including treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs).
研究概览
简要总结
This is the second in-human 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 assess the safety, tolerability, ciliary rescue, pharmacodynamic biomarkers, and preliminary efficacy of RCT1100 following multiple doses of inhaled RCT1100 administered via nebulizer to participants with Primary Ciliary Dyskinesia caused by disease-causing 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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Healthy, adult, male or female of, 18-70 years of age, inclusive, at screening.
- •Participant has clinical diagnosis of PCD and disease-causing mutations in the DNAI1 gene
- •Participant has a forced expiratory volume in one second (FEV1) of at least 50% 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 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
The number of participants with Adverse Events (AEs), including treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs).
时间窗: From Baseline Through Week 24
Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs), as well as an adverse event of special interest (AESI): "Fever", which will include body temperature and any associated symptoms (chills, myalgia).
次要结局
未报告次要终点