NCT06344026Enrolling By Invitation1 期
Phase 1/2a Dose Escalation Study of ANPD001 in Sporadic Parkinson Disease
Aspen Neuroscience18 个研究点 分布在 1 个国家目标入组 9 人开始时间: 2024年1月23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
- Enrolling By Invitation
- 发起方
- 入组人数
- 9
- 试验地点
- 18
- 主要终点
- Incidence and severity of treatment emergent adverse events (Safety and Tolerability)
研究概览
简要总结
This clinical trial is designed to test the safety and tolerability of injecting ANPD001 cells that will mature into dopamine-producing cells into the brain of participants with Parkinson Disease. All participants will have ANPD001 cells manufactured from their own previously collected cells.
详细描述
Participants will undergo a surgical implantation of cells that will mature into dopamine-producing neurons under general anesthesia into a part of the brain where dopamine production is decreased in patients with Parkinson Disease. The effect on Parkinson Disease symptoms, safety and tolerability, and cell survival are assessed for 5 years post-transplant (with MRI and PET imaging scans of the brain). Safety and tolerability are assessed annually for an additional 10 years via telephone call (total follow-up of 15 years)
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Age 40-75 years of age at time of consent in the trial-ready cohort study ANPD001-01
- •Met all eligibility requirements for inclusion in the trial-ready cohort in clinical study ANPD001-01
- •Diagnosed with Parkinson Disease at least 4 years ago
- •Unequivocal motor response to Levodopa
排除标准
- •Prior brain surgery that, in the opinion of the neurologist or neurosurgeon, contraindicates administration of ANPD001
- •History of intracranial therapy for PD, including deep brain stimulation (DBS), focused ultrasound (FUS), gene therapy or other biological therapy
- •History of cognitive impairment or dementia
- •History of clinically significant Dopa Dysregulation syndrome
- •History of epilepsy, stroke, multiple sclerosis, poorly controlled or progressive neurological disease (other than PD), or poorly controlled cardiovascular disease
- •Inability to temporarily stop anticoagulation or antiplatelet therapy for at least 2 weeks
- •History of malignancy (cerebral or systemic) within the prior 5 years, except treated cutaneous squamous or basal cell carcinomas
- •Contraindication to MRI / contrast CRT and/or use of contrast agents
- •Weight > 300 lbs or Body Mass Index (BMI) > 35
- •Newly diagnosed diabetes (HbA1c > 7.0%), uncontrolled diabetes > 8.0% requiring frequent medication changes, or any other acute or chronic medical condition that would significantly increase the risks of a neurosurgical procedure
- •Pregnancy or lactation
- •Significant drug-induced dyskinesia (> 3 for any body part on the Abnormal Involuntary Movement Scale [AIMS])
- •Male or female with reproductive capacity who is unwilling to use barrier contraception for 3 months post-administration of the investigational product
- •Unable to comply with the protocol procedures, including frequent and prolonged follow-up assessments
- •Any significant issue raised by the neurologist or neurosurgeon
研究组 & 干预措施
ANPD001
Experimental
干预措施: Custom Device (Device)
ANPD001
Experimental
干预措施: ANPD001 (Biological)
结局指标
主要结局
Incidence and severity of treatment emergent adverse events (Safety and Tolerability)
时间窗: 1 year (primary follow up)
incidence and severity of treatment emergent adverse events (TEAE) and serious adverse events (SAE)
次要结局
- "ON" time without troublesome dyskinesia(1 year (primary follow up) and 5 years (long term follow up))
- Incidence and severity of treatment emergent adverse events during long term follow-up (Continued Safety and Tolerability)(14 years of long term follow up (4 years in-person visits and 10 additional years follow up via telephone call))
- Post-injection change in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II (Activities of Daily Living - ADL) and Part III (motor score) in the ON state (total score and scores for Parts I-IV)(1 year (primary follow up) and 5 years (long term follow up))
- Post-injection change in the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score in the practically defined OFF state(1 year (primary follow up) and 5 years (long term follow up))
- Post-injection change in the 18F-DOPA uptake in the putamen(1 year (primary follow up) and 5 years (long term follow up))
研究者
研究点 (18)
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相关资讯
Aspen Neuroscience's Personalized Parkinson's Cell Therapy Shows Promise in Phase II Trial, Advances to Pivotal Study- Aspen Neuroscience's autologous stem cell therapy sasineprocel demonstrated improved motor function and quality of life in eight Parkinson's disease patients after 12 months of treatment.
- The therapy showed mean improvements in "ON" time of 2.1-2.4 hours and significant reductions in MDS-UPDRS motor scores, with no serious surgical adverse events observed.
- Based on these encouraging Phase Ia/II results, the company plans to advance sasineprocel to a Phase III pivotal trial later this year.
- The personalized approach converts patients' own skin cells into dopamine-producing neurons, potentially avoiding immunosuppression required by off-the-shelf alternatives.6 months agoAspen Neuroscience's ANPD001 Shows Promise in Early Parkinson's Trial• Aspen Neuroscience has completed the first two cohorts in its ASPIRO Phase 1/2a trial for Parkinson's disease, evaluating ANPD001.
• ANPD001, an autologous dopaminergic neuronal cell replacement therapy, has demonstrated good tolerability with no serious adverse events reported to date.
• The ASPIRO trial is assessing the safety and tolerability of escalating doses of ANPD001 in patients with moderate to severe Parkinson's.
• Aspen is advancing the program to investigate a new commercial formulation of ANPD001, marking a significant step in autologous cell therapy for Parkinson's.last year