NCT06526923招募中1 期
A Single Ascending Dose, Phase 1/2 Trial to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SP-101 Via Nebulizer for the Treatment of Cystic Fibrosis (CF)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 15
- 试验地点
- 8
- 主要终点
- Incidence and severity of adverse events
研究概览
简要总结
This is a Phase 1/2 multicenter, open-label, single dose trial of SP-101 investigational gene therapy in adults with CF who are ineligible for or intolerant to CFTR modulator therapy.
详细描述
This multi-center study is a first-in-human, single ascending dose, Phase 1/2 trial to evaluate the safety, pharmacokinetics, and pharmacodynamics of various dose levels in people with CF who are ineligible or intolerant to CFTR modulator therapy.
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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or females, age 18 to 65 years at Screening Visit, inclusive
- •Diagnosis of CF
- •ppFEV1 value between 50-100% (inclusive)
- •Resting oxygen saturation ≥94% on room air by pulse oximetry 5 . Clinically stable CF disease as assessed by the Investigator and not requiring any new class of interventional treatment within the last 3 months prior to Screening
排除标准
- •Any change in established pulmonary treatment (including antibiotics) within 28 days prior to Screening Visit. However, inhaled beta-agonists can be included within 2 weeks prior to Screening Visit.
- •Clinically significant episode of hemoptysis (>50 mL or ¼ cup or 10 teaspoons per day) within 12 weeks prior to dosing with study drug on Day 1
- •Lung infection with Mycobacterium abscessus associated with a more rapid decline in pulmonary status
- •Currently receiving treatment for active lung infection with Burkholderia cenocepacia or Burkholderia dolosa
- •History of solid organ or hematological transplantation
- •History of clinically significant cirrhosis with or without portal hypertension
- •History of pulmonary hypertension
- •History of cardiotoxicity, a history of known coronary artery disease, and/or existing cardiomyopathy
- •Current active fungal infection (not just a positive culture), acute blood, lung, or bladder infection, clinically significant hepatic or renal dysfunction, and/or viral infection (including human immunodeficiency virus or hepatitis virus B or C) requiring the initiation of new therapy within 30 days prior to Screening
- •History of allergic bronchopulmonary aspergillosis (ABPA)
- •Uncontrolled diabetes mellitus, as evidenced by hemoglobin A1c >9% at Screening
- •Clinically significant laboratory abnormalities at Screening
- •Subjects with any medical condition or abnormal laboratory result that, in the opinion of the Investigator, will interfere with the safe completion of the study
- •Subjects who received any investigational products within 30 days (or 5 therapeutic half-lives, whichever is longer) prior to Screening
- •Subjects who have previously received any gene therapy agent
- •Subjects with known sensitivity to SP-101, doxorubicin or its excipients
结局指标
主要结局
Incidence and severity of adverse events
时间窗: 52 weeks
Safety and tolerability of SP-101 following a single inhalation dose, as assessed by incidence and severity of treatment emergent adverse events, serious adverse events, and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters.
次要结局
未报告次要终点
研究者
研究点 (8)
Loading locations...
相似试验
已完成
1 期
A Study to Assess the Pharmacokinetics and Safety of CSL312 in Healthy Japanese and Caucasian AdultsHealthy VolunteersNCT04580654CSL Behring38
已完成
1 期
A Safety and Tolerability Study of CTP-730 in Healthy VolunteersHealthyNCT02239081Concert Pharmaceuticals55
终止
1 期
Single Dose Safety, Tolerability and Pharmacokinetic Study of NCTX in Healthy VolunteersHealthy VolunteersNCT02063594Marval Pharma Ltd.20
Unknown
1 期
A Safety, Tolerability, and Dermal Reactogenicity Study of PDC-APBContact DermatitisNCT04853823Hapten Sciences, Inc.32
已完成
1 期
A Phase I, Single-Center Study of PDC-APB in Healthy VolunteersContact DermatitisNCT02700373Hapten Sciences, Inc.40