An Exploratory Clinical Trial of UX-GIP001 Injection (Human GABAergic Interneuron Progenitor Cells Injection) in Adult Patients With Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Incidence and severity of Adverse Events (AEs)/Serious Adverse Events (SAEs)
研究概览
简要总结
This is an exploratory clinical trial (Protocol: UX-GIP001-101) investigating UX-GIP001 Injection, a novel cell therapy product consisting of human GABAergic interneuron progenitor cells (GIP), for treating adult patients with drug-resistant unilateral medial temporal lobe epilepsy (MTLE). The primary objective is to assess the safety, tolerability, and preliminary efficacy of UX-GIP001. This is an open-label, single-arm study. All enrolled participants will receive the active investigational cell therapy. Seizure frequency and safety parameters will be evaluated by comparing post-transplant outcomes to pre-transplant baselines.
详细描述
Participants will receive UX-GIP001 via stereotactic neurosurgery, preceded by and followed with immunosuppressive therapy. The study includes a baseline period for eligibility confirmation and a 24-month follow-up phase involving regular safety assessments, neuroimaging , seizure diary logging, and evaluations of quality of life, cognition, and mood. This pioneering regenerative approach seeks to provide a new treatment strategy for drug-resistant MTLE by addressing the underlying pathophysiology.
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 18-75 (inclusive), male or female.
- •Clinical presentation consistent with unilateral mesial temporal lobe epilepsy (MTLE).
- •Has failed to achieve seizure control despite treatment with at least two anti-seizure medications (ASMs) at recommended or maximally tolerated doses.
- •Average focal seizure frequency ≥4 per 28 days during the 3 months prior to screening.
- •Stable doses of ASMs for ≥1 month prior to enrollment.
- •Patient is in good general health or has stable comorbid conditions, and has adequate organ function.
排除标准
- •Epilepsy caused by other/or progressive neurological diseases , or patients experiencing only focal aware seizures without observable manifestations.
- •History of epilepsy surgery.
- •History of status epilepticus within 12 months prior to screening.
- •Presence of long-term implants in the skull or intracranial space.
- •Severe systemic disease or dysfunction.
- •Primary or secondary immunodeficiency.
- •History of clear suicidal intent, plan, or behavior within one year prior to screening.
- •Severe psychiatric disorders.
- •History of malignancy within the past 5 years, except for cervical carcinoma in situ, basal cell or squamous cell skin cancer cured for >5 years.
- •Pregnant or breastfeeding women.
研究组 & 干预措施
UX-GIP001
Two dose levels will be planned. Each patient only receives one corresponding dose of UX-GIP001.
干预措施: UX-GIP001 (Biological)
结局指标
主要结局
Incidence and severity of Adverse Events (AEs)/Serious Adverse Events (SAEs)
时间窗: From baseline to 2 years post surgery
次要结局
- Change from baseline in total and subtype seizure frequency(From baseline to 2 years post surgery)
- Responder rates (≥50% and ≥75% reduction) in total and subtype seizure frequency(From baseline to 2 years post surgery)
- Seizure free rate(From baseline to 2 years post surgery)