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

NCT07572812 | Not Yet Recruiting Phase 1

• 1 other identifier: UX-GIP001-102
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Epilepsy

Keywords

Epilepsy; MTLE; Cell therapy

Outcome Measures

Primary Outcomes (1)

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

  From baseline to 6 Months post-treatment

Secondary Outcomes (5)

• 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

Study Arms (1)

UX-GIP001

EXPERIMENTAL

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

Drug: UX-GIP001

Interventions

UX-GIP001 DRUG

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 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.

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

• Shanghai UniXell Biotechnology Co., Ltd: lead
• West China Hospital: collaborator
• Beijing Tiantan Hospital: collaborator
• The First Affiliated Hospital of Zhengzhou University: collaborator

Study Sites (3)

Beijing Tiantan Hospital, Capital Medical University

Beijing, Beijing Municipality, 100070, China

Location

The First Affiliated Hospital of Zhengzhou University

Zhengzhou, Henan, 450000, China

Location

West China School of Medicine and West China Hospital, Sichuan University

Chengdu, Sichuan, 610041, China

Location

MeSH Terms

Conditions

Epilepsy

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xianwei Zhi

CONTACT

+86-21-60197620; xianweizhi@unixell.com

Study Design

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

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

Study Record Dates

• First Submitted: April 17, 2026
• First Posted: May 7, 2026
• Study Start (Estimated): May 20, 2026
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029
• Last Updated: May 7, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (3)