NCT07636811招募中2 期
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study, to Evaluate Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
Alnylam Pharmaceuticals5 个研究点 分布在 2 个国家目标入组 58 人开始时间: 2026年8月17日最近更新:
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 58
- 试验地点
- 5
研究概览
简要总结
The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 40 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21
- •Positive amyloid PET scan
- •Cognitively stable in the opinion of the investigator
- •Seizures must be well controlled with no occurrence of seizures in the 6 months prior to screening
排除标准
- •Has severe intellectual disability (ID)
- •Has a history of DS regression disorder
- •Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2×upper limit of normal (ULN) at Screening
- •Has estimated glomerular filtration rate (eGFR) <45 mL/min/1.73m^2 at Screening
- •Has recently received an investigational agent
- •Has had treatment with amyloid-targeting antibody
- •Comorbidities such as obstructive sleep apnea and hypothyroidism that are not well controlled
- •Note: other protocol defined inclusion / exclusion criteria apply
研究组 & 干预措施
Mivelsiran (Treatment Group B)
Experimental
Participants will be administered multiple high doses of mivelsiran.
干预措施: Mivelsiran (Drug)
Mivelsiran (Treatment Group A)
Experimental
Participants will be administered multiple low doses of mivelsiran.
干预措施: Mivelsiran (Drug)
Placebo (Treatment Group C)
Placebo Comparator
Participants will be administered multiple doses of placebo.
干预措施: Placebo (Drug)
研究者
研究点 (5)
Loading locations...
相似试验
招募中
3 期
Evaluation of efficacy and safety of early in hospital initiation of inclisiran treatment in patients with acute coronary syndromes2025-521670-34-00Novartis Pharma AG24
招募中
3 期
Coronary Computed Tomography Study to Assess the Effect of Inclisiran in Addition to Maximally Tolerated Statin Therapy on Atherosclerotic Plaque Progression in Participants With a Diagnosis of Non-obstructive Coronary Artery Disease Without Previous Cardiovascular Events (V-PLAQUE)2024-511126-31-00Novartis Pharma AG173
招募中
3 期
A Study to Evaluate Tobevibart + Elebsiran in Chronic HDV Infection2024-515919-22-00Vir Biotechnology Inc.11
招募中
4 期
A study to evaluate the safety and effectiveness of a cholesterol-lowering injection of Inclisiran Sodium in Indian patients with high cholesterol or mixed lipid disorders.CTRI/2024/10/075998Novartis Healthcare Pvt Ltd65
招募中
3 期
A study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged 1 to less than 12 years with hemophilia A or B.Hemophilia2025-521858-42-00Sanofi-Aventis Recherche & Developpement2