NCT07662096招募中1 期
A First-In-Human Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of ARO-033 in Adult Participants
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 84
- 试验地点
- 1
- 主要终点
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
This study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of ARO-033 compared to placebo in adult normal healthy volunteers (NHVs) and the safety, tolerability, PK, PD, and efficacy of multiple doses of ARO-033 in participants with age-related macular degeneration (AMD).
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- 是
入选标准
- •NHV cohorts only: Adults 18 to 65 years of age who are not pregnant, not breastfeeding, and do not plan to become pregnant (or impregnate their partners) during the study and for at least 90 days following the end of the study.
- •AMD cohorts only: Adults ≥50 years of age who are not pregnant, not breastfeeding, and do not plan to become pregnant (or impregnate their partners) during the study and for at least 90 days following the end of the study.
- •Body mass index (BMI) between 18.0 and 35.0 kilograms (kg)/square meter (m^2), inclusive.
- •No abnormal finding of clinical relevance at the Screening evaluation that in the opinion of the Investigator could adversely impact participant's safety during the study or adversely impact study results.
- •AMD cohorts only: A clinical diagnosis of AMD in both eyes as determined by the Investigator and confirmed by the central reading center.
排除标准
- •All Cohorts:
- •Human immunodeficiency virus (HIV) infection, as shown by the presence of anti-HIV antibody (seropositive).
- •Seropositive for hepatitis B virus (HBV) (hepatitis B surface antigen positive at screening) or hepatitis C virus (HCV) (HCV antibody positive with reflex confirmation using HCV RNA amplification at Screening). Cured HCV (positive antibody test without detectable HCV RNA) is permitted if HCV RNA has been negative for at least 2 years.
- •Uncontrolled hypertension (resting systolic blood pressure ≥160 millimeters of mercury [mmHg] or diastolic blood pressure ≥95 mmHg, confirmed by repeat measurement, at Screening).
- •Evidence of clinically significant immunocompromising condition (for example, primary immunodeficiency syndrome, aplastic anemia, known or suspected complement factor deficiency, or any other condition resulting in significantly impaired immune response as evidenced by recurrent infections), or recent/ongoing treatment with immunosuppressive agents.
- •History of major surgery within 90 days of Screening.
- •Use of an investigational agent or device within 30 days or 5 half-lives (whichever is longer) prior to dosing or current participation in an investigational study. Participants recently participating in studies involving investigational agents with prolonged therapeutic effect (such as ribonucleic acid interference [RNAi] therapeutics, cell or gene therapies) and whose last dose was less than 12 months prior to the first dose of study intervention, should be discussed with the Medical Monitor.
- •Any medical condition or clinically significant laboratory abnormality at Screening that in the opinion of the Investigator should exclude the participant from participation, preclude safe and successful completion of the study, or confound study results.
- •AMD Cohorts Only:
- •Any ocular condition in the study eye that may be expected to progress and that may affect central vision or otherwise be a confounding factor during the study, as determined by the Investigator.
- •Atrophic retinal disease due to causes other than AMD (for example, drug-induced, Stargardt disease, cone rod dystrophy) in either eye.
- •Note: Other protocol-defined inclusion and exclusion criteria may apply.
研究组 & 干预措施
NHV Cohorts: ARO-033
Experimental
Participants will receive either a single dose of ARO-033 on Day 1 (single-ascending dose [SAD]) or 2 doses (multiple-ascending dose [MAD]) of ARO-033 administered on Day 1 and Day 29.
干预措施: ARO-033 (Drug)
NHV Cohorts: Placebo
Placebo Comparator
Participants will receive either a single dose of placebo on Day 1 (SAD) or 2 doses (MAD) of placebo administered on Day 1 and Day 29.
干预措施: Placebo (Drug)
AMD Cohorts: ARO-033
Experimental
Participants will receive up to 5 doses of ARO-033.
干预措施: ARO-033 (Drug)
AMD Cohorts: Placebo
Placebo Comparator
Participants will receive up to 5 doses of placebo.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
时间窗: Up to Day 337
次要结局
- Maximum Observed Plasma Concentration (Cmax) of ARO-033(SAD: Predose (0 hour) up to 48 hours postdose (Day 1 up to Day 3); MAD: Predose up to 18 hours postdose (Days 1 and 29), 24 hours postdose (Days 2 and 30), and 48 hours postdose (Days 3 and 31); AMD cohorts: Up to 4 hours postdose (Days 1 and 29))
- Area Under the Plasma Concentration-time Curve From Time 0 to the Last Quantifiable Plasma Concentration (AUC0-t) of ARO-033(SAD: Predose (0 hour) up to 48 hours postdose (Day 1 up to Day 3); MAD: Predose up to 18 hours postdose (Days 1 and 29), 24 hours postdose (Days 2 and 30), and 48 hours postdose (Days 3 and 31); AMD cohorts: Up to 4 hours postdose (Days 1 and 29))
- NHV Cohorts Only: Amount of ARO-033 Excreted in the Urine From Time 0 to 24 Hours After Dosing (Ae)(SAD: Predose (0 hour) up to 8 hours postdose (Day 1), and up to 24 hours postdose (Day 2); MAD: Predose (0 hour) up to 8 hours postdose (Days 1 and 29))
研究者
研究点 (1)
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