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

NCT07244328 | Recruiting Early Phase 1

• 1 other identifier: 2025-0876
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Epilepsy; MTLE

Keywords

Unilateral Medial Temporal Lobe Epilepsy (MTLE); GABAergic Inhibitory Interneuron Progenitor Cells; Drug-Resistant Epilepsy (DRE)

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of Adverse Events (AEs)/Serious Adverse Events (SAEs)

  From baseline to 2 years post surgery

Secondary Outcomes (3)

• 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

Study Arms (1)

UX-GIP001

EXPERIMENTAL

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

Biological: UX-GIP001

Interventions

UX-GIP001 BIOLOGICAL

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

UX-GIP001

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Second Affiliated Hospital, School of Medicine, Zhejiang University: lead
• Shanghai UniXell Biotechnology Co., Ltd: collaborator

Study Sites (1)

Second Affiliated Hospital, School of Medicine, Zhejiang University

Hangzhou, Zhejiang, 310009, China

RECRUITING

MeSH Terms

Conditions

Epilepsy; Drug Resistant Epilepsy

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Shuang Wang, Doctor

  Second Affiliated Hospital, School of Medicine, Zhejiang University

  PRINCIPAL INVESTIGATOR

• Junming Zhu, Doctor

  Second Affiliated Hospital, School of Medicine, Zhejiang University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yang, Doctor

CONTACT

+86 132 0571 6189; 12418578@zju.edu.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: open-label, single-center, dose-escalation and dose-expansion

Study Record Dates

• First Submitted: November 17, 2025
• First Posted: November 24, 2025
• Study Start: November 10, 2025
• Primary Completion (Estimated): November 30, 2028
• Study Completion (Estimated): November 30, 2028
• Last Updated: April 22, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)