A Phase I Clinical Trial of UX-GIP001 in the Treatment of Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

Официальное название

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

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат UX-GIP001
  Allogeneic human GABAergic interneuron precursors (UX-GIP001) are delivered into the temporal lobe region of the brain.

Первичные конечные точки

• Incidence and severity of Adverse Events (AEs)/Serious Adverse Events (SAEs) [Срок оценки: From baseline to 6 Months post-treatment]

Вторичные конечные точки (5)

• Change from baseline in total and subtype seizure frequency [Срок оценки: From baseline to 2 years post-treatment]
• Responder rates (≥50% and ≥75% reduction) in total and subtype seizure frequency [Срок оценки: From baseline to 2 years post-treatment]
• Seizure free rate [Срок оценки: From baseline to 2 years post-treatment]
• Incidence and severity of AEs/SAEs related to surgery, transplanted cells, and/or immunosuppressive therapy [Срок оценки: From baseline to 2 years post-treatment]
• Incidence and severity of all AEs/SAEs [Срок оценки: From baseline to 2 years post-treatment]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 3 центра

• Beijing Tiantan Hospital, Capital Medical University — Пекин
• The First Affiliated Hospital of Zhengzhou University — Чжэнчжоу
• West China School of Medicine and West China Hospital, Sichuan University — Чэнду

Идентификаторы

NCT: NCT07572812 · UX-GIP001-102

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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