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.
연구 개요
상세 설명
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.
연구 유형
등록 (추정된)
단계
- 1단계
연락처 및 위치
연구 연락처
- 이름: Xianwei Zhi
- 전화번호: +86-21-60197620
- 이메일: xianweizhi@unixell.com
연구 장소
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Beijing Municipality
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Beijing, Beijing Municipality, 중국, 100070
- 모병
- Beijing Tiantan Hospital, Capital Medical University
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연락하다:
- Xiaoqiu Shao
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Henan
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Zhengzhou, Henan, 중국, 450000
- 아직 모집하지 않음
- The First Affiliated Hospital of Zhengzhou University
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연락하다:
- Qun Wang
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Sichuan
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Chengdu, Sichuan, 중국, 610041
- 모병
- West China School of Medicine and West China Hospital, Sichuan University
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연락하다:
- Dong Zhou, Doctor
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
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.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
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실험적: 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. |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
기간 |
|---|---|
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Incidence and severity of Adverse Events (AEs)/Serious Adverse Events (SAEs)
기간: From baseline to 6 Months post-treatment
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From baseline to 6 Months post-treatment
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2차 결과 측정
결과 측정 |
기간 |
|---|---|
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Change from baseline in total and subtype seizure frequency
기간: 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
기간: 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
기간: 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
기간: 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
기간: From baseline to 2 years post-treatment
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From baseline to 2 years post-treatment
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공동 작업자 및 조사자
협력자
수사관
- 수석 연구원: Dong Zhou, Doctor, West China Hospital
- 수석 연구원: Xiaoqiu Shao, Doctor, Beijing Tiantan Hospital
- 수석 연구원: Qun Wang, Doctor, The First Affiliated Hospital of Zhengzhou University
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
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