A Phase I Clinical Trial to Evaluate the Safety, Tolerability, and Preliminary Efficacy of UX-GIP001 (Human GABAergic Interneuron Progenitor Cells Injection) in the Treatment of Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 12
- 试验地点
- 3
研究概览
简要总结
This is a phase I study (Protocol: UX-GIP001-102) 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 years (inclusive), male or female;
- •Diagnosis of focal epilepsy according to the International League Against Epilepsy (ILAE) 2025 epilepsy classification, with disease duration ≥2 years;
- •Clinical presentation consistent with unilateral mesial temporal lobe epilepsy (MTLE);
- •Meeting the diagnostic criteria for drug-resistant epilepsy;
- •Average focal seizure frequency ≥4 per 28 days within the 3 months prior to screening;
- •On a stable dose of anti-seizure medications for ≥1 month prior to enrollment;
- •Patient has adequate organ function as follows: absolute neutrophil count ≥2.0×10⁹/L; white blood cell count ≥4.0×10⁹/L; platelet count ≥100×10⁹/L; AST and ALT ≤2.5× upper limit of normal (ULN); total bilirubin ≤1.5× ULN; serum creatinine ≤1.5× ULN; estimated glomerular filtration rate (eGFR) ≥60 mL/min/1.73 m² (calculated using the CKD-EPI formula);
- •Women of childbearing potential must have a negative serum pregnancy test during the screening period. All participants and their partners agree to have no plans for pregnancy, sperm donation, or egg donation from the time of signing the informed consent form until 2 years after cell transplantation treatment, and voluntarily adopt contraceptive measures deemed effective by the investigator;
- •Patient has good compliance, with a patient diary completion rate ≥80% prior to enrollment.
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 (Drug)