A Phase I/II Safety and Dose Evaluation Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral Idiopathic Parkinson's Disease (SUNRISE-PD)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 6
- 试验地点
- 3
- 主要终点
- Safety of OXB-102 as measured by changes in vital signs
研究概览
简要总结
This study consists of two parts. Part A will evaluate the safety and tolerability of multiple doses of OXB-102 (AXO-Lenti-PD) in participants with Parkinson's disease. Part B will assess the safety and efficacy of the selected dose of OXB-102 in participants with Parkinson's disease.
详细描述
This study consists of two parts. Part A is an open-label dose-escalation phase in which participants are enrolled in cohorts and will receive one of approximately three escalating doses of OXB-102 (AXO-Lenti-PD). Part B is a randomized, double-blind phase in which participants will be randomized to either an active group receiving the selected dose from Part A, or to a control group receiving an imitation surgical procedure (ISP).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Study Part A open-label and Study Part B randomized, double-blind
入排标准
- 年龄范围
- 30 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with bilateral idiopathic PD
- •Males/females between 30 and 70 years at the time of surgery
- •Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) score of between 30 and 60 in the "OFF" medication state
- •Presence of motor fluctuations and/or dyskinetic movement
- •Candidate for surgical intervention
- •Hoehn and Yahr (H&Y) Stage 3 or 4 in the "OFF" medication state
- •Stable dosing of PD medication, including L-DOPA, for four weeks prior to screening with Levodopa equivalent daily dose (LEDD) of at least 900 mg
排除标准
- •History of psychosis or current treatment with dopamine blocking agents and prior regular exposure to antipsychotic agents
- •History of stereotactic or other surgery for the treatment of PD, including Deep Brain Stimulation (DBS)
- •Participation in a prior cell or gene transfer therapy study
- •Contraindications to use of anaesthesia
- •Current or anticipated treatment with anticoagulant therapy or the use of anticoagulation therapy that cannot be temporarily stopped around the time of surgery
- •Diagnosis of multiple system atrophy
- •Abnormal MRI findings such as mega cisterna, septum pellucidum, signs of severe cortical or subcortical atrophy, brain tumours, vascular diseases, trauma or arteriovenous malformations
- •Presence of dementia
研究组 & 干预措施
OXB-102 Dose Level 1
OXB-102 Dose Level 1 Single Administration (Part A: open-label)
干预措施: OXB-102 (Drug)
OXB-102 Dose Level 2
OXB-102 Dose Level 2 Single Administration (Part A: open-label)
干预措施: OXB-102 (Drug)
OXB-102 Dose Level 3
OXB-102 Dose Level 3 Single Administration (Part A: open-label)
干预措施: OXB-102 (Drug)
OXB-102 Selected Dose
Selected Dose of OXB-102 Single Administration (Part B: double-blind)
干预措施: OXB-102 (Drug)
Imitation Surgical Procedure
General anesthesia with bilateral skin incisions (Part B: double-blind)
干预措施: Imitation Surgical Procedure (ISP) (Other)
结局指标
主要结局
Safety of OXB-102 as measured by changes in vital signs
时间窗: 3 months timepoint
Number of clinically significant changes in vital signs
Safety of OXB-102 as measured by changes in clinical laboratory analysis
时间窗: 3 months timepoint
Number of clinically significant changes in clinical laboratory analysis
Safety of OXB-102 as measured by changes in brain MRI findings
时间窗: 3 months timepoint
Number of clinically significant changes in brain MRI findings
Safety of OXB-102 as measured by incidence of treatment emergent adverse events and serious adverse events
时间窗: 3 months timepoint
Treatment emergent adverse events and serious adverse events will be assessed by National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE), version 4.0, for severity
Safety of OXB-102 as measured by changes in physical examination
时间窗: 3 months timepoint
Number of clinically significant changes in physical examination
次要结局
- Change in Unified Parkinson's Disease Rating Scale (UPDRS) scores defined in "OFF" and "ON" medication states(Baseline to 6 months)
- Change in dyskinesia rating scale score(Baseline to 6 months)
- Change in "OFF" time during waking day compared to baseline as assessed by participant diaries(Baseline to 6 months)