NCT07836452招募中1 期
A Phase 1, Randomized, Double-Masked, Placebo-Controlled, Single-Ascending and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CITY-RBP4 Administered Subcutaneously in Healthy Volunteers and Participants With Stargardt Disease Type 1 (STGD1)
City Therapeutics1 个研究点 分布在 1 个国家目标入组 48 人开始时间: 2026年9月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 48
- 试验地点
- 1
- 主要终点
- Incidence, severity, and relationship of treatment-emergent adverse events (TEAEs)
研究概览
简要总结
This is a first-in-human (FIH), multicenter, randomized, double-masked, placebo-controlled adaptive clinical study evaluating the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) of CITY-RBP4, following single-ascending doses and multiple doses, via subcutaneous (SC) administration.
详细描述
The study consists of 3 sequential parts:
- Part A: Single-ascending doses in healthy volunteers
- Part B: Single-ascending doses in participants with Stargardt Disease Type 1 (STGD1)
- Part C: Multiple doses in participants with Stargardt Disease Type 1 (STGD1)
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
- 12 Years 至 45 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Willing and able to provide written informed consent form
- •Be aged 18 to 45 years (inclusive) at the time of signing the informed consent form
- •Generally healthy on medical history, exam, labs, and ECG
- •BCVA 20/25 or better in each eye
- •Serum retinol above protocol threshold
- •Body weight ≥40 kg and BMI of 18.0 to 32.0 kg/m2
- •Men and WOCBP must agree to use highly effective methods of contraception or practice abstinence from 30 days before the first dose of CITY-RBP4, throughout the study, and for at least 180 days after treatment with CITY-RBP4 or the last study visit, whichever is longer; men must refrain from donating or banking sperm during the same period
- •Female participants who are not WOCBP should meet at least 1 of the following criteria: have undergone a documented hysterectomy, bilateral oophorectomy, and/or bilateral salpingectomy; have medically confirmed ovarian failure, or achieved postmenopausal status
排除标准
- •Clinically significant ocular disease or prior ocular surgery that could interfere with safety assessments
- •History of vitamin A deficiency, malabsorption, or chronic diarrhea
- •Clinically significant cardiac, hepatic, renal, pulmonary, neurologic, or psychiatric disease
- •Positive hepatitis B, hepatitis C, or HIV screening
- •Prior RNAi/oligonucleotide therapy or recent investigational treatment
- •Recent vitamin A-related supplements, retinoids, or medications affecting vitamin A metabolism
- •Pregnancy or breastfeeding, if applicable
- •Key Inclusion Criteria (Parts B and C)
- •Participants aged 12 to 45 years
- •Clinical diagnosis of STGD1 with documented ABCA4 pathogenic/likely pathogenic variant
- •Minimum BCVA is required in the study eye
- •Subjects must have a defined aggregate atrophic lesion in 1 or both eyes
- •Body weight ≥40 kg
- •Able to complete key ophthalmic safety assessments and comply with study precautions
- •Key Exclusion Criteria (Parts B and C)
- •Ocular conditions that could confound retinal imaging or safety evaluation
- •Prior ocular gene therapy, stem cell therapy, or retinal implant
- •Vitamin A deficiency or recent prohibited vitamin A-related treatments
- •Recent interventional study participation
- •Clinically significant non-ophthalmic disease, abnormal screening labs/ECG, or positive hepatitis B, hepatitis C, or HIV screening
- •Pregnancy or breastfeeding, if applicable
研究组 & 干预措施
CITY-RBP4 in Healthy Adults (Part A)
Experimental
干预措施: CITY-RBP4 (Drug)
In Healthy Adults (Part A)
Placebo Comparator
干预措施: Placebo (Drug)
CITY-RBP4 in Participants with STGD1 (Parts B & C)
Experimental
干预措施: CITY-RBP4 (Drug)
In Participants with STGD1 (Parts B & C)
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Incidence, severity, and relationship of treatment-emergent adverse events (TEAEs)
时间窗: Through study completion, up to Day 360
To evaluate the safety and tolerability of single and multiple doses of CITY-RBP4 in healthy adults and participants with Stargardt disease Type 1 (STGD1)
次要结局
- Area under plasma concentration-time curve (AUC)(Day 1 to Day 2)
- Plasma concentrations of CITY-RBP4(Day 1 to Day 2)
- Urine concentrations of CITY-RBP4(Part A and B only: Day 1 to Day 2)
- Maximum plasma concentration (Cmax)(Day 1 to Day 2)
- Time to maximum plasma concentration (Tmax)(Day 1 to Day 2)
- Terminal elimination half-life (t1/2)(Day 1 to Day 2)
- Apparent clearance (CL/F)(Day 1 to Day 2)
- Amount excreted unchanged (Ae)(Part A and B only: Day 1 to Day 2)
- Change from baseline in plasma RBP4 concentrations(Up to Day 360)
- Change from baseline in serum retinol concentrations(Up to Day 360)
研究者
研究点 (1)
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