NCT05994534进行中（未招募）1 期
A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Concentration of Cystine Levels Over Time
研究概览
简要总结
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
详细描述
This study will examine the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients, aged ≥ 10 years. The ability of NPI-001 to reduce cystine will be assessed and compared with cysteamine.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or females, any race, ≥ 10 years of age.
- •Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days.
- •Females will be nonpregnant and nonlactating, and females of childbearing potential and males will agree to use contraception as detailed in the protocol.
- •Able to comprehend and willing to sign an informed consent /assent form and to abide by the study restrictions (travel as necessary, clinical phase 1 unit or similar for up to 3 days).
排除标准
- •Have undergone kidney transplantation.
- •Are receiving dialysis treatment.
- •History of significant hypersensitivity to NAC or any ingredient of NPI-001 oral solution.
- •Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the 30 days prior to Day
- •Inability to provide blood samples, including difficulty with venous access.
- •Subjects who, in the opinion of the Investigator and/or Sponsor (or designee), should not participate in this study.
研究组 & 干预措施
NPI-001
Experimental
Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.
干预措施: N-Acetylcysteine Amide (Drug)
cysteamine
Active Comparator
Single dose, tablets in current treatment dose
干预措施: Cysteamine Bitartrate (Drug)
结局指标
主要结局
Concentration of Cystine Levels Over Time
时间窗: 1 day
Cystine concentration over 6 hours
次要结局
未报告次要终点
研究者
研究点 (1)
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