The Safety, Tolerability and Efficacy of NouvNeu001 for Parkinson's Disease

Официальное название

A Phase I/II Study to Assess the Safety, Tolerability and Efficacy of NouvNeu001 Injection for Mid- to Late-stage Parkinson's Disease

Обзор

This clinical trial is designed to evaluate the safety, tolerability and preliminary efficacy of a single injection of NouvNeu001 (Human Dopaminergic Progenitor Cells Injection) in patients with Parkinson's disease.

Подробное описание

This is a multi-center, single arm, and open label trial. The NouvNeu001 will be transplanted into bilateral putamen/striatum using stereotactic neurosurgery. Subjects will take immunosuppressants to prevent potential immune rejection for 24 to 36 weeks.

Вмешательства

• Биопрепарат Human Dopaminergic Progenitor Cells
  Single injection of Human Dopaminergic Progenitor Cells into the putamen/striatum region of brain.

Первичные конечные точки

• Safety and Tolerability [Срок оценки: 24 weeks and 48 weeks post-transplant]
• Motor Function [Срок оценки: 24 weeks post-transplant]

Вторичные конечные точки (2)

• The Motor Function and Non-motor Function [Срок оценки: 48 weeks and 96 weeks post-transplant]
• Continued Safety and Tolerability [Срок оценки: 96 weeks and 5 years post-transplant]

Критерии участия

Критерии включения

• Age 50-75 years old, male or female
• Able to understand the rationale of the clinical trial and sign informed consent form (ICF)
• Diagnosis of Parkinson's Disease in accordance with the MDS clinical diagnostic criteria for Parkinson's disease (2015)
• Diagnosis of Parkinson's Disease made between 4 to 20 years ago
• Medically suitable for neurosurgery under anesthesia and able to participate in Magnetic Resonance Imaging (MRI) and Positron Emission Tomography (PET) scan.
• Hoehn-Yahr staging for "off" episodes is 2.5 to 4
• The MDS-UPDRS Part III (MDS-UPDRS-III) score in the "off" state > 35, and positive for the Acute Levodopa Challenge Test (ALCT)
• Acceptable laboratory test results during screening and prior to transplantation

Критерии исключения

• Atypical Parkinsonism
• Patients who have had previous pallidotomy, deep brain stimulation (DBS) surgery, striatal or extrapyramidal surgery, brain stereotaxy or other brain surgery; as well as other surgical procedures that are judged by the investigator to affect patient's 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 with a history of severe cardiovascular and cerebrovascular diseases
• Patients with a history of malignant tumors
• Patients who have had previous cellular therapy
• Patients with active disseminated intravascular coagulation and significant hemorrhagic tendency within 3 months prior to screening, or who cannot temporarily suspend anti-platelet agents or other anti-coagulant medications for at least 5 days before surgery
• Patients with long-term, heavy use of glucocorticoids or immunosuppressive drugs within 3 months prior to signing the ICF
• Patients with a history of mental illness who are deemed unfit to participate in the study by the investigator; or a history of suicidal ideation or suicide attempts within the past year or currently;
• Patients who have used botulinum toxin within 6 months prior to signing the ICF
• Patients with active epilepsy or currently on anti-epileptic drugs
• Patients with a history of dementia or severe cognitive disorder, or the score of MDS-UPDRS 1.1 during screening is > 3; poor compliance, inability to accurately keep diary, and/or inability to sign ICF due to dementia
• Patients with severe depression or with severe anxiety during screening;
• Patients with the following abnormalities during screening, including: Abnormal coagulation; Abnormal immunological tests, and assessed by the investigator it is not suitable to participate in the trial; Hypertensive patients with poorly controlled blood pressure and patients with severe postural hypotension; Diabetic patients with poorly controlled blood glucose
• Patients with surgical contraindications, or with 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)
• Presence of one of the following: positive for human immunodeficiency virus (HIV) antibody, treponema pallidum antibody, hepatitis C virus (HCV) antibody and HCV RNA; Hepatitis B virus (HBV) surface antigen positive and HBV DNA copy number > detection of normal values; Tuberculosis is in the active stage; Other active infections that the investigator believes may affect Patients' participation in the study or affect study outcomes;
• Patients with alcohol addiction or positive for drug of abuse testing
• Patients with a history of contraindication or allergy to the drugs used during the study or any of its components, or are allergic to the same drugs or other macrolides, or have allergies;
• Female of childbearing potential who are not surgically sterilized/premenopausal/unwilling to use medically approved effective contraception with 2 years after administration of investigational drug and lactating women; men who are not surgically sterilized/unwilling to use medically approved effective contraception with 2 years after administration of investigational drug
• Patients who have received electric shock therapy within 30 days prior to surgery
• Patients who are participating in other clinical trials, or have been enrolled in other clinical studies and received intervention therapy within 3 months prior to the surgery
• Patients with poor compliance based on clinical evaluation of the investigator
• Patients who are being treated with drugs such as apomorphine, or levodopa/carbidopa infusion therapy
• Patients with severe dyskinesia in both on- and off-drug states

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 2 центра

• Beijing Hospital — Пекин
• Zhongnan Hospital of Wuhan University — Ухань

Идентификаторы

NCT: NCT06167681 · NouvNeu001-02

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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