NCT06285643招募中2 期
A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- AskBio Inc
- 入组人数
- 127
- 试验地点
- 85
- 主要终点
- Change from baseline to Month 18 on PD Motor Diary
研究概览
简要总结
The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.
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
- 45 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent Type of Subject and Disease Characteristics
- •Diagnosed with Parkinson's disease in the past 4-10 years (inclusive) as defined by the following:
- •Presence of bradykinesia PLUS any of the following:
- •Rest tremor
- •Postural instability
- •Presence of motor fluctuations as measured by the PD Motor Diary
- •Stable anti-parkinsonian medication regimen for >/= 4 weeks prior to screening
- •Must demonstrate responsiveness to levodopa therapy
排除标准
- •Known history or current evidence of medical, genetic, or neurological conditions that may provide an alternative to idiopathic PD diagnosis
- •Presence or history of significant vascular and/or cardiovascular disease
- •Presence of significant cognitive impairment, poorly controlled depression/anxiety
- •Presence or history of psychosis or impulse control disorder
- •History of malignancy other than treated cutaneous squamous or basal cell carcinomas
- •Presence of clinically relevant conditions that could compromise surgical suitability and/or subject safety
- •Contraindication to magnetic resonance imaging and/or use gadolinium-based contrast agents
- •Prior history of brain surgery including, but no limited: DBS, pallidotomy focused ultrasound thalamotomy, or other experimental neurosurgical procedure
- •Chronic immunosuppressive therapy
研究组 & 干预措施
Control Surgery
Sham Comparator
干预措施: control surgery (Procedure)
AAV2-GDNF
Experimental
干预措施: AAV2-GDNF gene therapy (Drug)
结局指标
主要结局
Change from baseline to Month 18 on PD Motor Diary
时间窗: 18 months
Normalized Good ON time per Hauser Diary
Change from baseline to Month 18 on PD Motor Diary
时间窗: 18 months
Normalized Good ON time per Hauser Diary
次要结局
未报告次要终点
研究者
研究点 (85)
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相关资讯
AskBio Advances Commercial-Scale Manufacturing for Parkinson's Gene Therapy in Phase II Trial- AskBio has introduced its commercial manufacturing process to supply ametefgene parvec (AB-1005) for the REGENERATE-PD Phase II trial following FDA IND amendment approval.
- The investigational gene therapy delivers GDNF protein to the brain via adeno-associated viral vector to potentially slow Parkinson's disease progression and improve motor outcomes.
- REGENERATE-PD is enrolling 127 participants aged 45-75 with moderate-stage Parkinson's disease across sites in Germany, Poland, the United Kingdom, and the United States.
- The therapy has received multiple regulatory designations including FDA Fast Track, RMAT designation, and Japan's SAKIGAKE status for Parkinson's disease treatment.5 months agoFive Promising Therapeutic Candidates in Parkinson's Disease Clinical Pipeline- Ambroxol, a repurposed cough suppressant, shows potential as a disease-modifying therapy for Parkinson's by enhancing lysosomal function and clearing α-synuclein aggregates, with the GREAT trial currently evaluating its efficacy.
- Novel anti-inflammatory compounds targeting the NLRP3 inflammasome, including Inzomelid and NT-0796, are advancing through clinical trials as potential disease-modifying agents that could slow neurodegeneration in Parkinson's disease.
- Gene therapy approach AAV2-GDNF and innovative non-dopaminergic treatments like Solangepras and Glovadalen represent significant advances in the therapeutic pipeline, offering potential for durable neuroprotection and improved symptom management with fewer side effects.last year