Epilepsy, MTLE
Conditions
Keywords
Unilateral Medial Temporal Lobe Epilepsy (MTLE), GABAergic Inhibitory Interneuron Progenitor Cells, Drug-Resistant Epilepsy (DRE)
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.
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
BIOLOGICALUX-GIP001
Allogeneic human GABAergic interneuron precursors (UX-GIP001) are delivered into the temporal lobe region of the brain.
Sponsors
Second Affiliated Hospital, School of Medicine, Zhejiang University
Shanghai UniXell Biotechnology Co., Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
open-label, single-center, dose-escalation and dose-expansion
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of Adverse Events (AEs)/Serious Adverse Events (SAEs) | From baseline to 2 years post surgery |
Secondary
| 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 |
Countries
China
Contacts
CONTACTYang, Doctor
PRINCIPAL_INVESTIGATORShuang Wang, Doctor
Second Affiliated Hospital, School of Medicine, Zhejiang University
PRINCIPAL_INVESTIGATORJunming Zhu, Doctor
Second Affiliated Hospital, School of Medicine, Zhejiang University
Outcome results
None listed