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

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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Epilepsy
• Intervention / Treatment: Drug: 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): 12
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Xianwei Zhi; Phone Number: +86-21-60197620; Email: xianweizhi@unixell.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100070
      • Beijing Tiantan Hospital, Capital Medical University
      • Contact: Xiaoqiu Shao
  • Henan
    • Zhengzhou, Henan, China, 450000
      • The First Affiliated Hospital of Zhengzhou University
      • Contact: Qun Wang
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • West China School of Medicine and West China Hospital, Sichuan University
      • Contact: Dong Zhou, Doctor

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

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.	Drug: 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 6 Months post-treatment

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Shanghai UniXell Biotechnology Co., Ltd
• Collaborators: Beijing Tiantan Hospital; West China Hospital; The First Affiliated Hospital of Zhengzhou University
• Investigators: Principal Investigator: Dong Zhou, Doctor, West China Hospital; Principal Investigator: Xiaoqiu Shao, Doctor, Beijing Tiantan Hospital; Principal Investigator: Qun Wang, Doctor, The First Affiliated Hospital of Zhengzhou University

Study record dates

Study Major Dates

• Study Start (Estimated): May 20, 2026
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029

Study Registration Dates

• First Submitted: April 17, 2026
• First Submitted That Met QC Criteria: April 30, 2026
• First Posted (Actual): May 7, 2026

Study Record Updates

• Last Update Posted (Actual): May 7, 2026
• Last Update Submitted That Met QC Criteria: April 30, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Epilepsy; Cell therapy; MTLE
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy
• Other Study ID Numbers: UX-GIP001-102

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