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.
Studie Overzicht
Gedetailleerde beschrijving
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.
Studietype
Inschrijving (Geschat)
Fase
- Fase 1
Contacten en locaties
Studiecontact
- Naam: Xianwei Zhi
- Telefoonnummer: +86-21-60197620
- E-mail: xianweizhi@unixell.com
Studie Locaties
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Beijing Municipality
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Beijing, Beijing Municipality, China, 100070
- Werving
- Beijing Tiantan Hospital, Capital Medical University
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Contact:
- Xiaoqiu Shao
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Henan
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Zhengzhou, Henan, China, 450000
- Nog niet aan het werven
- The First Affiliated Hospital of Zhengzhou University
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Contact:
- Qun Wang
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Sichuan
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Chengdu, Sichuan, China, 610041
- Werving
- West China School of Medicine and West China Hospital, Sichuan University
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Contact:
- Dong Zhou, Doctor
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Beschrijving
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.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
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Experimenteel: UX-GIP001
Two dose levels will be planned. Each patient only receives one corresponding dose of UX-GIP001. |
Allogeneic human GABAergic interneuron precursors (UX-GIP001) are delivered into the temporal lobe region of the brain. |
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
|---|---|
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Incidence and severity of Adverse Events (AEs)/Serious Adverse Events (SAEs)
Tijdsspanne: From baseline to 6 Months post-treatment
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From baseline to 6 Months post-treatment
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Secundaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
|---|---|
|
Change from baseline in total and subtype seizure frequency
Tijdsspanne: From baseline to 2 years post-treatment
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From baseline to 2 years post-treatment
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Responder rates (≥50% and ≥75% reduction) in total and subtype seizure frequency
Tijdsspanne: From baseline to 2 years post-treatment
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From baseline to 2 years post-treatment
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Seizure free rate
Tijdsspanne: From baseline to 2 years post-treatment
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From baseline to 2 years post-treatment
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Incidence and severity of AEs/SAEs related to surgery, transplanted cells, and/or immunosuppressive therapy
Tijdsspanne: From baseline to 2 years post-treatment
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From baseline to 2 years post-treatment
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Incidence and severity of all AEs/SAEs
Tijdsspanne: From baseline to 2 years post-treatment
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From baseline to 2 years post-treatment
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Medewerkers en onderzoekers
Medewerkers
Onderzoekers
- Hoofdonderzoeker: Dong Zhou, Doctor, West China Hospital
- Hoofdonderzoeker: Xiaoqiu Shao, Doctor, Beijing Tiantan Hospital
- Hoofdonderzoeker: Qun Wang, Doctor, The First Affiliated Hospital of Zhengzhou University
Studie record data
Bestudeer belangrijke data
Studie start (Werkelijk)
Primaire voltooiing (Geschat)
Studie voltooiing (Geschat)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- UX-GIP001-102
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
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