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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Epilepsy; MTLE
• Intervention / Treatment: Biological: UX-GIP001

Detailed 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.

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Yang, Doctor; Phone Number: +86 132 0571 6189; Email: 12418578@zju.edu.cn

Study Locations

• China
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310009
      • Recruiting
      • Second Affiliated Hospital, School of Medicine, Zhejiang University
      • Contact: Yang, Doctor; Phone Number: +86 132 0571 618; Email: 12418578@zju.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: UX-GIP001; Two dose levels will be planned. Each patient only receives one corresponding dose of UX-GIP001.	Biological: UX-GIP001; Allogeneic human GABAergic interneuron precursors (UX-GIP001) are delivered into the temporal lobe region of the brain.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence and severity of Adverse Events (AEs)/Serious Adverse Events (SAEs)	From baseline to 2 years post surgery

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Second Affiliated Hospital, School of Medicine, Zhejiang University
• Collaborators: Shanghai UniXell Biotechnology Co., Ltd
• Investigators: Principal Investigator: Shuang Wang, Doctor, Second Affiliated Hospital, School of Medicine, Zhejiang University; Principal Investigator: Junming Zhu, Doctor, Second Affiliated Hospital, School of Medicine, Zhejiang University

Study record dates

Study Major Dates

• Study Start (Actual): November 10, 2025
• Primary Completion (Estimated): November 30, 2028
• Study Completion (Estimated): November 30, 2028

Study Registration Dates

• First Submitted: November 17, 2025
• First Submitted That Met QC Criteria: November 17, 2025
• First Posted (Actual): November 24, 2025

Study Record Updates

• Last Update Posted (Actual): April 22, 2026
• Last Update Submitted That Met QC Criteria: April 17, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Unilateral Medial Temporal Lobe Epilepsy (MTLE); GABAergic Inhibitory Interneuron Progenitor Cells; Drug-Resistant Epilepsy (DRE)
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Drug Resistant Epilepsy; Epilepsy
• Other Study ID Numbers: 2025-0876

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No