NCT04931862终止1 期
A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 35
- 试验地点
- 17
- 主要终点
- Safety: Proportion of patients with adverse events (AEs)
研究概览
简要总结
This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of intrathecal (IT) WVE-004 in adult patients with C9orf72-associated ALS or FTD. To participate in the study, patients must have a documented mutation (GGGGCC [G4C2] repeat expansion) in the first intronic region of the C9orf72 gene and be diagnosed with ALS or FTD.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •ALS-specific: Diagnosis of ALS based on clinical manifestations.
- •ALS-specific: Clinically diagnosed possible, laboratory supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology revised El Escorial criteria.
- •ALS-specific: Patients receiving riluzole have been on a stable dose for a minimum of 30 days.
- •ALS-specific: Patients on edaravone have received a minimum of 1 cycle (28 days).
- •ALS-specific: Patients discontinuing riluzole or edaravone had the last dose administered ≥1 month prior to Screening.
- •FTD-specific: Must have Global Clinical Dementia Rating - Frontotemporal Lobar Degeneration (CDR® plus NACC FTLD) score of 0.5 or
- •FTD-specific: Able to undergo periodic magnetic resonance imaging (MRI) of the brain. Participants with mixed phenotype (ALS and FTD) need not undergo MRI if their ALS symptoms prevent it.
- •Mixed-phenotype: Patients who are mixed phenotype (ALS and FTD) must meet both the ALS-specific and FTD-specific criteria.
排除标准
- •Clinically significant medical finding on the physical examination other than C9orf72-associated ALS or FTD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the trial procedures
- •Received any other investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Received an investigational oligonucleotide, within the past 6 months or 5 half-lives of the drug, whichever is longer.
研究组 & 干预措施
WVE-004 (Dose C) or placebo
Experimental
干预措施: WVE-004 (Drug)
WVE-004 (Dose C) or placebo
Experimental
干预措施: Placebo (Drug)
WVE-004 (Dose A) or placebo
Experimental
干预措施: WVE-004 (Drug)
WVE-004 (Dose A) or placebo
Experimental
干预措施: Placebo (Drug)
WVE-004 (Dose B) or placebo
Experimental
干预措施: WVE-004 (Drug)
WVE-004 (Dose B) or placebo
Experimental
干预措施: Placebo (Drug)
WVE-004 (Dose D) or placebo
Experimental
干预措施: WVE-004 (Drug)
WVE-004 (Dose D) or placebo
Experimental
干预措施: Placebo (Drug)
结局指标
主要结局
Safety: Proportion of patients with adverse events (AEs)
时间窗: Period 1 Day 1 to Period 2 Week 24 (end of study)
次要结局
- Pharmacodynamic: Change from baseline in concentration of poly-GP levels in the CSF(Period 1 Day 1 to Period 2 Week 24 (end of study))
- Pharmacokinetic: Concentration of WVE-004 in cerebrospinal fluid (CSF)(Period 1 Day 1 to Period 2 Week 24 (end of study))
研究者
研究点 (17)
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