Epilepsy
Conditions
Keywords
Epilepsy, MTLE, Cell therapy
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.
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.
Interventions
DRUGUX-GIP001
Allogeneic human GABAergic interneuron precursors (UX-GIP001) are delivered into the temporal lobe region of the brain.
Sponsors
Shanghai UniXell Biotechnology Co., Ltd
West China Hospital
Beijing Tiantan Hospital
The First Affiliated Hospital of Zhengzhou University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
open-label, multi-center, dose-escalation and dose-expansion
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of Adverse Events (AEs)/Serious Adverse Events (SAEs) | From baseline to 6 Months post-treatment |
Secondary
| 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 |
Countries
China
Contacts
CONTACTXianwei Zhi
PRINCIPAL_INVESTIGATORDong Zhou, Doctor
West China Hospital
PRINCIPAL_INVESTIGATORXiaoqiu Shao, Doctor
Beijing Tiantan Hospital
PRINCIPAL_INVESTIGATORQun Wang, Doctor
The First Affiliated Hospital of Zhengzhou University
Outcome results
None listed