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The Safety,Tolerability, Efficacy of XS411 Cell Injection for the Treatment of Primary Parkinson's Disease

2026년 9월 28일 업데이트: XellSmart Therapeutics (Shanghai) Co., Ltd.

A Phase I/II Clinical Study on the Safety, Tolerability, and Efficacy of Stereotactic Intracerebral Transplantation of Human Allogeneic iPSC-derived Dopaminergic Neuronal Precursor Cells (XS411 Cell Injection) for the Treatment of Parkinson's Disease

This Phase I clinical trial is designed to evaluate the safety, tolerability of a single dose of XS411 (derived dopaminergic neural progenitor cell injection) in patients with primary Parkinson's disease.

This Phase II clinical trial is designed to evaluate the safety, tolerability and Efficacy of XS411 (derived dopaminergic neural progenitor cell injection) in patients with Parkinson's disease.

연구 개요

상태

모집하지 않고 적극적으로

정황

개입 / 치료

상세 설명

This is a phase I clinical study to evaluate the safety and tolerability of human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection (XS411CN) via brain stereotactic transplantation for the treatment of primary Parkinson's disease.

The study adopts a single-arm, open-label, non-randomized dose-escalation design to evaluate the safety, tolerability, and preliminary efficacy of stereotactic brain transplantation of XS411CN in patients with primary Parkinson's disease and explore the optimal recommended dose. 12 to 18 patients with primary Parkinson's disease are planned to be enrolled.

On the day of cell transplantation surgery, the planned dose of XS411CN is transplanted into the bilateral putamen of the subjects by brain stereotactic technology.

After receiving the XS411CN transplantation, the subjects will receive immunosuppressants. All subjects receive standard treatment at the same time.

Each patient receives a safety observation period of 28 days after cell transplantation with XS411CN. This study adopts the traditional "3+3 " dose escalation method. The trial starts with a low dose and the dose will be increased gradually. Each subject will be closely observed for 7 days after surgery, and the dose-limiting toxicity (DLT) events that may occur within 28 days after the first transplant treatment will be evaluated.

연구 유형

중재적

등록 (실제)

39

단계

  • 2 단계
  • 1단계

연락처 및 위치

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연구 장소

    • Beijing Municipality
      • Beijing, Beijing Municipality, 중국, 100070
        • Beijing Tiantan Hospital

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

50년 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

Inclusion Criteria:

  1. Male or female, aged between 50 and 75 years old;
  2. Diagnosed as 'clinically established PD' consistent with MDS clinical Diagnostic Criteria for Parkinson's disease,
  3. A minimum of 5 years since diagnosis;
  4. Hoehn- Yahr score during the off-time is 3 to 4;
  5. The subjects with an average of at least 2.5 hours of "off time" per day;
  6. Stable anti-PD therapy for at least 1 month before screening;
  7. Anti-PD treatment was once effective, but the efficacy has declined significantly or drug-induced motor complications have occurred, affecting the quality of life;
  8. Positive levodopa challenge test (MDS-UPDRS-III score improvement from off- state to on- state > 30%);
  9. At least three months before the first administration, the subject should have completed all vaccinations as recommended by local authorities;
  10. The results of complete blood count meet the following conditions: ANC ≥ 2.0×109 /L, WBC ≥ 4.0×109 /L, PLT≥ 100×109 /L, HGB ≥ 10g/dL
  11. The patient or his/her legal guardian agree to accept the study and signed the informed consent form in writing.

Exclusion Criteria:

  1. Parkinsonian syndrome or secondary Parkinson's disease (Atypical Parkinson's syndrome (Parkinson's plus syndrome, secondary Parkinson's syndrome, hereditary degenerative Parkinson's syndrome);

  2. Patients are in advanced stages of Parkinson's disease and are experiencing severe, disabling peak-dose dyskinesia or biphasic dyskinesia and/or unpredictable or widely fluctuating symptoms;

  3. Severe cognitive impairment (MMSE<24, poor compliance due to dementia, inability to accurately record diaries, and/or inability to sign informed consent;

  4. Those with a history of severe mental illness, or those with severe suicidal tendencies as measured by the Columbia-Suicide Severity Rating Scale (C-SSRS), or those with suicidal ideation in the past 12 months, or those who have attempted suicide in the past 5 years;

  5. Patients with severe brain atrophy, space-occupying lesions, hydrocephalus, cerebral vascular malformations as indicated by head CT/MRI, and those with structural abnormalities that may affect transplantation or increase surgical risks as determined by the investigators;

  6. Currently with active internal bleeding; or arterial puncture at a site that is difficult to stop bleeding within 1 week before screening; or gastrointestinal or urinary bleeding within 3 weeks before screening;

  7. History of striatal or extrapyramidal surgery, including but not limited to deep brain stimulation (DBS) surgery and globus pallidus lesion;

  8. Coagulation abnormalities;

  9. Abnormal liver and kidney function laboratory tests during the screening period;

  10. The patient is currently receiving or has previously received the following treatments:

    • Use of benzodiazepines at a dose level equal to or higher than the recommended dose in Package Insert for more than 4 weeks cumulatively since one month before enrollment, immunosuppressants (except for the immunosuppressants to be used in this study), or antipsychotics within 3 months before treatment;
    • Use of botulinum toxin, phenol, subarachnoid baclofen injection, or interventional treatment for dystonia or spasticity within 6 months before treatment;
    • Have a history of epilepsy or use anti-epileptic drugs for prevention;
    • Received cell therapy within 3 months before screening;
  11. Contraindications to general anesthesia or stereotactic surgery (such as sleep apnea, chronic obstructive pulmonary disease, etc.), MRI or PET examinations;

  12. Those with the following abnormalities in the past or detected (if any of the following is met, they will be excluded):

    • Evidence of congestive heart failure or history of end-stage cardiovascular disease;
    • Severe arrhythmias and poorly controlled arrhythmias;
    • Clinically significant electrocardiogram abnormalities during the screening or baseline period;
    • History of unstable angina or acute myocardial infarction within the past 3 months;
    • Diabetic patients with poor glycemic control;
    • Hypertensive patients with poorly controlled blood pressure;
  13. Patients with active infection during the screening period (except mild local skin infection) that need antibiotic, antifungal, or antiviral treatment but infection is still not under control;

  14. The presence of severe lung diseases during the screening period;

  15. Patients with uncontrolled autoimmune diseases;

  16. During the screening period, patients have severe arthritis, limp, severe sequelae of stroke, severe osteoporosis, or history of severe trauma within 1 month, etc., which the investigators assess may affect the evaluations;

  17. Any history of malignancy, excluding cured basal cell carcinoma and/or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, or ductal carcinoma of the breast;

  18. Serious infectious diseases;

  19. Pregnant or lactating women, women who plan to become pregnant during the study and within 6 months after the end of the study, or women who are unwilling to use effective contraceptive measures;

  20. Patients who have participated in other clinical studies of drugs or medical devices within 3 months before treatment;

  21. Have a long history of alcoholism or drug abuse;

  22. Patients with a history of severe allergies or allergies to investigational drugs and immunosuppressants;

  23. Any other conditions that the investigator considers unsuitable for participation in the study.

  24. Severe depression

  25. Hallucinations, and hallucinations still exist after optimizing Parkinson's medications

  26. Patients have undergone MRI-guided focused ultrasound thalamotomy

  27. Used immunosuppressants within 3 months, except for the immunosuppressants to be used in this study

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위
  • 중재 모델: 병렬 할당
  • 마스킹: 하나의

무기와 개입

참가자 그룹 / 팔
개입 / 치료
간섭 없음: 표준 처리
실험적: 4.5 × 10^6 세포 / 양측 푸 타멘

5.0×10^7 cells/mL, injection, single dose

실험적: 9.0 × 10^6 세포 / 양측 푸 타멘

5.0×10^7 cells/mL, injection, single dose

실험적: 1.8 × 10^7 세포 / 양측 푸 타멘

5.0×10^7 cells/mL, injection, single dose

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
To evaluate the safety and tolerability of XS411CN in treatment of primary Parkinson's disease
기간: 28 days after administration of XS411CN

To evaluate the safety and tolerability of XS411CN in treatment of primary Parkinson's disease through:

1)The occurrence and incidence of adverse events and serious adverse events (adverse event grading criteria are based on NCI-CTCAE V5.0) 28 days after administration of XS411CN; 2) Changes in vital signs, laboratory tests, 12-lead electrocardiogram, etc. compared with baseline after administration of XS411CN;

28 days after administration of XS411CN
MDS-UPDRS Part III score during the 'OFF' state
기간: Month 12

Change from baseline in MDS-UPDRS Part III (Motor Examination) score during the 'OFF' state at 12 months after administration of XS411 cell injection

Month 12

2차 결과 측정

결과 측정
측정값 설명
기간
MDS-UPDRS Part III score during the 'OFF' state
기간: Baseline, Month 3, Month 6, Month 9

Change from baseline in MDS-UPDRS Part III (Motor Examination) score during the 'OFF' state at 3 months, 6 months, 9 months after administration of XS411 cell injection

Baseline, Month 3, Month 6, Month 9

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

협력자

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2025년 8월 5일

기본 완료 (추정된)

2027년 7월 1일

연구 완료 (추정된)

2028년 7월 1일

연구 등록 날짜

최초 제출

2025년 7월 9일

QC 기준을 충족하는 최초 제출

2025년 7월 18일

처음 게시됨 (실제)

2025년 7월 23일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 9월 30일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 9월 28일

마지막으로 확인됨

2026년 5월 1일

추가 정보

이 연구와 관련된 용어

기타 연구 ID 번호

  • XS411-Allo-LOPD-CN1/2-P01

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

예

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .