The Safety,Tolerability, Efficacy of XS411 Cell Injection for the Treatment of Primary Parkinson's Disease
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
상태
정황
정황
개입 / 치료
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
연구 유형
등록 (실제)
등록
단계
단계
- 2 단계
- 1단계
연락처 및 위치
연구 장소
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Beijing Municipality
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Beijing, Beijing Municipality, 중국, 100070
- Beijing Tiantan Hospital
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Male or female, aged between 50 and 75 years old;
- Diagnosed as 'clinically established PD' consistent with MDS clinical Diagnostic Criteria for Parkinson's disease,
- A minimum of 5 years since diagnosis;
- Hoehn- Yahr score during the off-time is 3 to 4;
- The subjects with an average of at least 2.5 hours of "off time" per day;
- Stable anti-PD therapy for at least 1 month before screening;
- Anti-PD treatment was once effective, but the efficacy has declined significantly or drug-induced motor complications have occurred, affecting the quality of life;
- Positive levodopa challenge test (MDS-UPDRS-III score improvement from off- state to on- state > 30%);
- At least three months before the first administration, the subject should have completed all vaccinations as recommended by local authorities;
- 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
- The patient or his/her legal guardian agree to accept the study and signed the informed consent form in writing.
Exclusion Criteria:
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Parkinsonian syndrome or secondary Parkinson's disease (Atypical Parkinson's syndrome (Parkinson's plus syndrome, secondary Parkinson's syndrome, hereditary degenerative Parkinson's syndrome);
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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;
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Severe cognitive impairment (MMSE<24, poor compliance due to dementia, inability to accurately record diaries, and/or inability to sign informed consent;
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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;
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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;
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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;
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History of striatal or extrapyramidal surgery, including but not limited to deep brain stimulation (DBS) surgery and globus pallidus lesion;
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Coagulation abnormalities;
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Abnormal liver and kidney function laboratory tests during the screening period;
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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;
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Contraindications to general anesthesia or stereotactic surgery (such as sleep apnea, chronic obstructive pulmonary disease, etc.), MRI or PET examinations;
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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;
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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;
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The presence of severe lung diseases during the screening period;
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Patients with uncontrolled autoimmune diseases;
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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;
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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;
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Serious infectious diseases;
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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;
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Patients who have participated in other clinical studies of drugs or medical devices within 3 months before treatment;
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Have a long history of alcoholism or drug abuse;
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Patients with a history of severe allergies or allergies to investigational drugs and immunosuppressants;
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Any other conditions that the investigator considers unsuitable for participation in the study.
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Severe depression
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Hallucinations, and hallucinations still exist after optimizing Parkinson's medications
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Patients have undergone MRI-guided focused ultrasound thalamotomy
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Used immunosuppressants within 3 months, except for the immunosuppressants to be used in this study
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 하나의
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
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간섭 없음: 표준 처리
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실험적: 4.5 × 10^6 세포 / 양측 푸 타멘
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5.0×10^7 cells/mL, injection, single dose |
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실험적: 9.0 × 10^6 세포 / 양측 푸 타멘
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5.0×10^7 cells/mL, injection, single dose |
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실험적: 1.8 × 10^7 세포 / 양측 푸 타멘
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5.0×10^7 cells/mL, injection, single dose |
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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To evaluate the safety and tolerability of XS411CN in treatment of primary Parkinson's disease
기간: 28 days after administration of XS411CN
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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
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MDS-UPDRS Part III score during the 'OFF' state
기간: Month 12
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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
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2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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MDS-UPDRS Part III score during the 'OFF' state
기간: Baseline, Month 3, Month 6, Month 9
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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
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공동 작업자 및 조사자
협력자
협력자
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
기타 연구 ID 번호
기타 연구 ID 번호
- XS411-Allo-LOPD-CN1/2-P01
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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