NCT07102342招募中1 期
A Phase 1/2 Clinical Study to Evaluate the Safety, Tolerability, Feasibility, and Preliminary Efficacy of NouvNeu001 in Patients With Advanced Parkinson's Disease.
iRegene Therapeutics Co., Ltd.1 个研究点 分布在 1 个国家目标入组 5 人开始时间: 2026年1月12日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Safety and Tolerability
研究概览
简要总结
This is a phase 1/2 clinical study to evaluate the safety, tolerability, feasibility, and preliminary efficacy of NouvNeu001 in patients with advanced Parkinson's Disease.
详细描述
This is a multi-center, single arm, and open label trial. The NouvNeu001 will be transplanted into the bilateral putamen/striatum using stereotactic neurosurgery. Subjects will take immunosuppressants to prevent potential immune rejection for 24 to 36 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 30 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patient must be 30 to 75 years of age inclusive, at the time of signing the informed consent form (ICF).
- •Type of Patient and Disease Characteristics
- •Diagnosis of PD between past 4 to 20 years (meet MDS 2015 clinical diagnostic criteria for PD).
- •H-Y staging (Appendix III) for "OFF" episodes is 2.5-4.
- •MDS-UPDRS-III score > 35, and positive for the Acute levodopa challenge test (ALCT) (improvement > 30% for MDS UPDRS-III staging from OFF episodes to ON episodes) over two screening period visits. Baseline scores will be computed as the means of two screening period visits.
- •No significant change in UPDRS-III scores between the two visits during the screening period.
- •Patients who take stable doing of dopamine drug for at least 4 weeks prior to receiving the study drug.
- •No significant change in dose and dosing schedule of the prescribed medicines for PD during the screening period.
- •Medically suitable for neurosurgery under general anesthesia.
- •Acceptable laboratory test values during screening and prior to transplantation (Day 0):
- •Absolute neutrophil count ≥ 2.0 × 109/L
- •White blood cell count ≥ 4.0 × 109/L
- •Platelet count ≥ 100 × 109/L
- •Aspartate aminotransferase (AST) and alanine transaminase (ALT) ≤ 2.5 x upper limit of normal (ULN)
- •Total bilirubin ≤ 1.5 x ULN
- •Serum creatinine ≤ 1.5 x ULN
- •eGFR ≥ 50 mL/min/1.73 m2
- •Agree to postpone any additional neurosurgical procedures, including DBS, until completion of the 12-month follow-up study (unless recommended by a neurologist).
- •Agree not to participate in any other therapeutic intervention study within 24 months after neurosurgery.
- •Able to participate in all study visits and evaluations, including brain/spine MRI, CT and PET scan.
- •Weight • Body mass index (BMI) within the range of 18~32 kg/m2 (inclusive) with a minimum body weight of 45 kg at screening.
- •Sex and Contraceptive/Barrier Requirements
- •Contraceptive use by men and women for 12 months after neurosurgery should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •Informed Consent
- •Able to understand the rationale of the clinical trial and sign the ICF. Other Inclusion Criteria
- •Agree not to receive COVID-19 vaccine within 14 days before neurosurgery, and not to receive any other vaccine within 30 days after neurosurgery
排除标准
- •Medical Conditions
- •Atypical Parkinsonism (Parkinsonism-Plus syndrome, secondary parkinsonism, familial parkinsonism) Prior/Concomitant Therapy
- •Patients who have had previous pallidotomy, DBS surgery, striatal or extrapyramidal surgery, brain stereotaxy, prior surgical or radiation therapy to the brain or spinal cord, or other brain surgery; as well as other surgical procedures that, in the investigator's judgment, could interfere with participation in this study.
- •Patients who have a previous head CT/MRI examination showing cerebral trauma, vascular malformation, hydrocephalus, brain tumor, etc., and patients who have brain imaging abnormalities in the striatum or other brain areas leading to a significantly increased risk for surgery.
- •Patients who have had previous cellular therapy.
- •Patients who have used glucocorticoids for an extended period (≥14 days) and at high doses (equivalent to prednisone ≥ 20 mg/day or other glucocorticoids at equivalent doses) within 3 months prior to signing the ICF. (excluding topical treatment)
- •Patients who have used immunosuppressive drugs for an extended period (≥14 days) within 3 months prior to signing the ICF.
- •Patients who have used antipsychotics, such as antidepressants, antimanic drugs, etc. within 3 months prior to signing the ICF and are deemed by the investigator to potentially impact the study assessment.
- •Patients who have used botulinum toxin or other drugs for dystonia or muscle spasticity within 6 months prior to signing the ICF and are deemed by the investigator to potentially impact the study assessment.
- •Prior/Concurrent Clinical Study Experience
- •Patients who are participating in other clinical trials, or have been enrolled in other clinical studies within 3 months prior to the screening
- •Patients with poor compliance based on clinical evaluation of the investigator. Diagnostic Assessments
- •Patients with a history of dementia or a severe cognitive disorder; or those with obvious dementia or congnitive impairment detected during screening; MDS-UPDRS congnitive impairment score (section 1.1) > 3 point at screening; or patinets with poor compliance, inability to accurately keep diary, and/or inability to sign ICF due to dementia.
- •Severe depression as defined by HAMD ≥ 24 at screening.
- •Severe anxiety as defined by HAMA ≥ 29 at screening.
- •Patients with a history of psychiatric disorders that, in the investigator's opinion, make them unsuitable for participation; or patients with a history of suicidal ideation or suicide attempts within the past year or currently (including actual attempts, interrupted attempts, or failed attempts).
- •Patients with abnormal coagulation (prothrombin time ≥ 1.5 ULN, activated partial thromboplastin time ≥ 1.5 ULN), or abnormal INR at screening.
- •Patients cannot temporarily suspend anti-platelet agents or other anti-coagulant medications at least 5 days prior to investigational drug administration surgery.
- •Patients with active disseminated intravascular coagulation and significant hemorrhagic tendency within 3 months prior to signing the ICF.
- •Patients with current or history of following disease
- •severe heart failure (congestive heart failure of New York Heart Association Class II or above), unstable angina pectoris, and myocardial infarction
- •severe arrhythmia, including but not limited to second- or third-degree atrioventricular block, or prolonged QT interval.
- •Long QT Syndrome.
- •cardiovascular surgery (cardiac, vascular stent surgery, angioplasty).
- •stroke or transient ischemic attack within 3 months prior to signing the ICF, as determined by the investigator to be unsuitable to participate the study.
- •subarachnoid hemorrhage
- •primary mitochondrial disorder, multiple sclerosis, or other neurodegenerative diseases such as Alzheimer's disease
- •major vascular diseases (aortic aneurysm, aortic dissecting aneurysm, internal carotid artery stenosis), as determined by the investigator to be unsuitable to participate the study.
- •previous or current malignant tumors
- •immune disfunction, including autoimmune disease or immunocompromised state
- •traumatic brain injury with loss of consciousness and residue neurologic symptoms
- •active epilepsy or currently on anti-epileptic drugs
- •Hypertensive patients with poorly controlled blood pressure (defined as blood pressure above 160/100 mmHg despite antihypertensive drugs treatment) and patients with severe postural hypotension
- •Diabetic patients with poorly controlled blood glucose (glycosylated hemoglobin > 9.0%, or fasting plasma glucose (FPG) ≥ 11.1mmol/L)
- •Patients with clinically significant abnormalities in kidney or liver function at screening.
- •Patient with clinically significant abnormalities in immunological tests at screening
- •Patients with surgical contraindications (such as those with cochlear implant, cardiac pacemaker, cardiac defibrillator, stereotactic nucleus pallidotomy; Patients who have had unilateral or bilateral intraparenchymal implantation of cellular products, or other surgical procedures within 6 months before screening which, in the opinion of the investigator, have an impact on this trial; Patients with other neurosurgical contraindications)
- •Patients with other combined severe systemic diseases, such as pulmonary heart disease, moderate to severe asthma, severe chronic obstructive pulmonary disease (COPD) (FEV1% < 50%)
- •Patients with serious infections, requiring antibiotic treatment within 2 weeks prior to signing the ICF
- •Patients with any active infectious disease, including but not limited to positive for human immunodeficiency virus, SARS Covid-19 virus, syphilis antibody, quantitative HCV-RNA testing, quantitative HBV-DNA testing greater than normal range for test results, or untreated tuberculosis; any other active infection that, in the investigator's opinion, may affect the participant's ability to take part in the study or could impact study outcomes.
- •Other Exclusion Criteria
- •Patients with alcohol or drug addiction
- •Patients with contraindications or a history of allergy to any medicines used during the neurosurgical procedure.
- •Patients with contraindication or a history of allergy to any medicines used during the study, such as immunosuppressants, levodopa, etc, or any of their components; Patients who are allergic to similar drugs or other macrolides.
- •Female of childbearing potential who are not surgically sterilized/premenopausal/unwilling to use medically approved effective contraception during the study and lactating women; men who are not surgically sterilized/unwilling to use medically approved effective contraception during the study
- •Patients who have received electric shock therapy within 30 days prior to the investigational drug administration surgery
- •Patients who are participating in other clinical trials, or have been enrolled in other clinical studies within 3 months prior to the screening
- •Patients with poor compliance based on clinical evaluation of the investigator
- •Patients who are being treated with drugs such as neuroleptics, apomorphine, or levodopa/carbidopa infusion therapy
- •Patients with severe dyskinesia in both "ON" and "OFF" drug states
- 另有 2 项未显示
研究组 & 干预措施
NouvNeu001
Experimental
Single injection of Human Dopaminergic Progenitor Cells into the bilateral putamen/striatum regions of the brain.
干预措施: Human Dopaminergic Progenitor Cells (Biological)
结局指标
主要结局
Safety and Tolerability
时间窗: 24 weeks and 48 weeks post-transplant
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs).
次要结局
- The Motor Function and Non-motor Function(48 weeks and 96 weeks post-transplant)
- Continued Safety and Tolerability(96 weeks and 15 years post-transplant)
研究者
研究点 (1)
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