UX-GIP001 cell therapy for adults with drug-resistant unilateral mesial temporal lobe epilepsy
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 treatment will try a one-time transplant of GABAergic interneuron progenitor cells into the affected side of the brain to see if it reduces seizures in adults with drug-resistant unilateral mesial temporal lobe epilepsy.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 6 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | Second Affiliated Hospital, School of Medicine, Zhejiang University Academic / other |
| Locations | 1 site (Hangzhou, Zhejiang) |
| Trial ID | NCT07244328 on ClinicalTrials.gov |
What this trial studies
This open-label, single-arm early-phase study delivers UX-GIP001, a preparation of human GABAergic interneuron progenitor cells, via stereotactic neurosurgery with perioperative immunosuppression. All participants receive the active cell therapy and outcomes after transplant will be compared to each patient's pre-transplant baseline for seizure frequency and safety. The protocol includes a baseline eligibility period followed by a 24-month follow-up with regular safety assessments, neuroimaging, seizure diary logging, and evaluations of quality of life, cognition, and mood. The main goals are to characterize safety and tolerability and to gather preliminary evidence of seizure reduction.
Who should consider this trial
Good fit: Adults aged 18–75 with unilateral mesial temporal lobe epilepsy who have failed at least two anti-seizure medications, have an average of ≥4 focal seizures per 28 days, are on stable medication doses, and have no major contraindications are ideal candidates.
Not a fit: Patients with bilateral or progressive epilepsies, those with only focal aware seizures, prior epilepsy surgery, severe systemic disease, immunodeficiency, or intracranial implants are unlikely to benefit or are ineligible.
Why it matters
Potential benefit: If successful, this approach could reduce seizure frequency and improve quality of life by restoring local inhibitory circuits as an alternative to resective surgery.
How similar studies have performed: Preclinical animal studies of GABAergic interneuron progenitor transplantation have shown promise for reducing seizures, but human data are very limited and this approach remains early and experimental.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Hangzhou, Zhejiang
- Second Affiliated Hospital, School of Medicine, Zhejiang University — Hangzhou, Zhejiang, China (Recruiting)
Study contacts
- Principal investigator: Shuang Wang, Doctor — Second Affiliated Hospital, School of Medicine, Zhejiang University
- Study coordinator: Yang, Doctor
- Email: 12418578@zju.edu.cn
- Phone: +86 132 0571 6189
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.