NCT06659640进行中（未招募）1 期
InsigHHT: A Phase 1/2, Randomized, Double-blind, Placebo-controlled, 2-part Study of the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Single Dose ALN-6400 in Adult Healthy Volunteers and Multiple Dose ALN-6400 in Adult Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
Alnylam Pharmaceuticals16 个研究点 分布在 6 个国家目标入组 89 人开始时间: 2024年11月7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
- 进行中（未招募）
- 发起方
- 入组人数
- 89
- 试验地点
- 16
- 主要终点
- Part A: Frequency of Adverse Events
研究概览
简要总结
The purpose of this study is to:
- evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers
- evaluate the efficacy, safety, tolerability and PD of multiple doses of ALN-6400 in adult patients with HHT
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Is a healthy adult volunteer
- •Is an adult patient with a clinical diagnosis of HHT
排除标准
- •Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN)
- •Has known human immunodeficiency virus (HIV) infection; or known current or chronic hepatitis C virus or hepatitis B virus infection
- •Has an estimated glomerular filtration (eGFR) of <90 mL/min/1.73m^2 at screening
- •Has ALT or AST >2×ULN
- •Has total bilirubin >1.5×ULN
- •Has eGFR of <30 mL/min/1.73m^2 at screening
- •Parts A and B:
- •Is not willing to comply with the contraceptive requirements during the study period
- •Note: other protocol defined inclusion / exclusion criteria apply
研究组 & 干预措施
Part B: Placebo
Placebo Comparator
Participants will be administered multiple doses of placebo.
干预措施: Placebo (Drug)
Part B: ALN-6400
Experimental
Participants will be administered multiple doses of ALN-6400.
干预措施: ALN-6400 (Drug)
Part A: ALN-6400
Experimental
Participants will be administered a single dose of ALN-6400.
干预措施: ALN-6400 (Drug)
Part A: Placebo
Placebo Comparator
Participants will be administered a single dose of placebo.
干预措施: Placebo (Drug)
结局指标
主要结局
Part A: Frequency of Adverse Events
时间窗: Up to Week 36
Part B: Frequency of Adverse Events
时间窗: Up to Week 96
次要结局
- Part A: Concentrations of ALN-6400 in Plasma(Predose and up to 2 days postdose)
- Part B: Change from Baseline in Plasminogen (PLG) in Plasma Protein Levels(Screening and up to Week 96 postdose)
- Part A: Change from Baseline in Plasminogen (PLG) in Plasma Activity Levels(Predose and up to Week 36 postdose)
- Part B: Change from Baseline in Plasminogen (PLG) in Plasma Activity Levels(Part B: Screening and up to Week 96 postdose)
- Part B: Change from Baseline in Epistaxis Frequency(Baseline up to Week 96)
- Part B: Change from Baseline in Iron Infusions(Baseline up to Week 96)
- Part A: Change from Baseline in Plasminogen (PLG) in Plasma Protein Levels(Predose and up to Week 36 postdose)
- Part B: Change from Baseline in Intensity-adjusted Epistaxis Duration(Baseline up to Week 96)
- Part B: Change from Baseline in Epistaxis Severity Score (ESS) Scale(Baseline up to Week 96)
- Part B: Change from Baseline in Epistaxis Duration(Baseline up to Week 96)
- Part B: Change from Baseline in Epistaxis Intensity(Baseline up to Week 96)
- Part B: Change from Baseline in Epistaxis-free Days per Month(Baseline up to Week 96)
- Part B: Change from Baseline in Hematologic Support Score (HSS)(Baseline up to Week 96)
- Part B: Change from Baseline in Red Blood Cell (RBC) Infusions(Baseline up to Week 96)
- Part B: Change from Baseline in Hemoglobin(Baseline up to Week 96)
- Part B: Change from Baseline in Quality of Life Patient-reported Outcomes (QoL/PRO) assessed by Nasal Outcome Score for Epistaxis in Hereditary Hemorrhagic Telangiectasia (NOSE HHT) Score(Baseline up to Week 84)
- Part B: Change from Baseline in QoL/PRO assessed by Modified Patient Global Impression of Severity (mPGI-S) Score(Baseline up to Week 84)
研究者
研究点 (16)
Loading locations...
相似试验
已完成
1 期
A Study to Evaluate ALN-AGT01 in Patients With HypertensionHypertensionNCT03934307Alnylam Pharmaceuticals124
已完成
1 期
Study of SGB-3908 in Healthy Subjects and Mildly Hypertensive SubjectsHypertension,EssentialNCT06501586Suzhou Sanegene Bio Inc.40
已完成
1 期
A Phase 1 Dose Escalation Study of VX-993 in Healthy ParticipantsPainNCT05653323Vertex Pharmaceuticals Incorporated70
已完成
1 期
A Phase 1 Study Evaluating the Safety, Pharmacokinetics, and Pharmacodynamics, of VX-993PainNCT06226454Vertex Pharmaceuticals Incorporated39
已完成
1 期
A Phase 1 Dose Escalation Study of VX-973 in Healthy AdultsPainNCT05866055Vertex Pharmaceuticals Incorporated30