NCT05835895进行中（未招募）1 期
A Phase 1b, Randomized, Double-Blinded, Placebo-Controlled Dose Ranging Study to Evaluate Safety, Tolerability and Pharmacodynamics of a Single Intra-articular Injection of GNSC-001 Gene Therapy in Subjects With Osteoarthritis of the Knee
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）
- 入组人数
- 67
- 试验地点
- 9
- 主要终点
- Severity of TEAEs and SAEs
研究概览
简要总结
The goal of this clinical study is to determine if a single injection of 1 or more dose levels of adeno-associated virus (AAV) gene therapy (GNSC-001) is safe and tolerated compared to placebo in participants with painful osteoarthritis (OA) of the knee.
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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •OA in at least one knee (the 'target knee'), confirmed using centrally read X-ray with a Kellgren-Lawrence (KL) score of 2 or 3
- •Western Ontario & McMaster University Osteoarthritis Index (WOMAC) pain score ≥ 20 (on a 50-point numerical rating scale) in the target knee during the seven days prior to Screening
- •Persistent moderate to severe symptoms in the target knee, despite failure or intolerance of a 3-month trial of at least 2 conservative therapies (e.g., activity modification, weight loss, physical therapy, opioids, and anti-inflammatory medications, and injection of hyaluronic acid or steroids)
- •Successful extraction of synovial fluid at Screening
- •Negative pregnancy test at Screening and Baseline (subjects of childbearing potential [SOCBP])
排除标准
- •Current or documented history of inflammatory arthritis (such as gout or rheumatoid arthritis), inflammation of connective tissue, or other immunological disease
- •Any form of joint degeneration as part of another syndrome (e.g., Ehler's Danlos, Stickler syndrome, etc.)
- •Surgery in the target knee within the 6 months prior to screening
- •Previous partial or total joint replacement in target knee
- •Significant and clinically evident malalignment of the target knee that would impact the subject's function, as determined by the Investigator
- •Body mass index (BMI) > 38 kg/m2
- •Clinically significant abnormal laboratory values at Screening in laboratory parameters that in the opinion of the Investigator, could represent a potential risk to participant safety:
- •Plans to have, elective surgery of other joints or spine or intra-articular injections in the target knee, within the next 12 months
研究组 & 干预措施
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
GNSC-001 (low dose)
Experimental
干预措施: GNSC-001 (Genetic)
GNSC-001 (low dose) + transient immune-modulation (oral)
Experimental
干预措施: GNSC-001 (Genetic)
GNSC-001 (low dose) + transient immune-modulation (oral)
Experimental
干预措施: transient immune-modulation (Drug)
GNSC-001 (high dose)
Experimental
干预措施: GNSC-001 (Genetic)
GNSC-001 (high dose) + transient immune-modulation (oral)
Experimental
干预措施: GNSC-001 (Genetic)
GNSC-001 (high dose) + transient immune-modulation (oral)
Experimental
干预措施: transient immune-modulation (Drug)
GNSC-001 (high dose) + transient immune-modulation (oral + injectable)
Experimental
干预措施: GNSC-001 (Genetic)
GNSC-001 (high dose) + transient immune-modulation (oral + injectable)
Experimental
干预措施: transient immune-modulation (Drug)
结局指标
主要结局
Severity of TEAEs and SAEs
时间窗: Month 12
Incidence of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
时间窗: Month 12
次要结局
- Synovial fluid Interleukin-1 Receptor Antagonist (IL-1Ra) levels(Months 1, 3, 6, and 24 months)
- Change in synovial Interleukin-1 Receptor Antagonist (IL-1Ra) levels over time(Baseline to Months 1, 3, 6, 12 and 24)
研究者
研究点 (9)
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