Exploratory Study of UX-GIP001 Cell Therapy in Adult Patients With Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)

Официальное название

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

Обзор

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.

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

• Биопрепарат 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 2 years post surgery]

Вторичные конечные точки (3)

• Change from baseline in total and subtype seizure frequency [Срок оценки: From baseline to 2 years post surgery]
• Responder rates (≥50% and ≥75% reduction) in total and subtype seizure frequency [Срок оценки: From baseline to 2 years post surgery]
• Seizure free rate [Срок оценки: From baseline to 2 years post surgery]

Критерии участия

Критерии включения

• 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.

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 · 1 центр

• Second Affiliated Hospital, School of Medicine, Zhejiang University — Ханчжоу

Идентификаторы

NCT: NCT07244328 · 2025-0876

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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