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

Zhejiang University

Status and phase: Not yet enrolling; Early Phase 1

Conditions: Epilepsy; MTLE

Treatments: Biological: UX-GIP001

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT07244328

2025-0876

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 6 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. Age 18-75 (inclusive), male or female.
2. Clinical presentation consistent with unilateral mesial temporal lobe epilepsy (MTLE).
3. Has failed to achieve seizure control despite treatment with at least two anti-seizure medications (ASMs) at recommended or maximally tolerated doses.
4. Average focal seizure frequency ≥4 per 28 days during the 3 months prior to screening.
5. Stable doses of ASMs for ≥1 month prior to enrollment.
6. Patient is in good general health or has stable comorbid conditions, and has adequate organ function.

Key Exclusion Criteria:

1. Epilepsy caused by other/or progressive neurological diseases , or patients experiencing only focal aware seizures without observable manifestations.
2. History of epilepsy surgery.
3. History of status epilepticus within 12 months prior to screening.
4. Presence of long-term implants in the skull or intracranial space.
5. Severe systemic disease or dysfunction.
6. Primary or secondary immunodeficiency.
7. History of clear suicidal intent, plan, or behavior within one year prior to screening.
8. Severe psychiatric disorders.
9. 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.
10. Pregnant or breastfeeding women.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

UX-GIP001

Experimental group

Description:

Two dose levels will be planned. Each patient only receives one corresponding dose of UX-GIP001.

Treatment:

Biological: UX-GIP001