The Safety, Tolerability and Preliminary Efficacy of NouvNeu001 for Early-onset Parkinson's Disease

NCT ID: NCT06608355

Last Updated: 2024-11-07

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-08-23
• Study Completion Date: 2027-03-31

Brief Summary

The goal of this clinical trial is to learn about the safety of NouvNeu001 injection in Early-onset Parkinson's Disease (EOPD) patients. It will also learn about the effects of NouvNeu001 treatment. The main questions it aims to answer are:

What medical problems do participants have when transplanting NouvNeu001 into bilateral putamen using stereotactic neurosurgery? Does injection of NouvNeu001 improve the motor function and non-motor function in participants?

Participants will:

Be injectioned the NouvNeu001 into bilateral putamen using stereotactic neurosurgery.

Take immunosuppressants to prevent potential immune rejection for 24 to 36 weeks.

Conditions

Early-onset Parkinson's Disease

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

NouvNeu001

Group Type: EXPERIMENTAL

Human Dopaminergic Progenitor Cells

Intervention Type: BIOLOGICAL

Single injection of Human Dopaminergic Progenitor Cells into the putamen of brain.

Interventions

Human Dopaminergic Progenitor Cells

Single injection of Human Dopaminergic Progenitor Cells into the putamen of brain.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Age 18-70 years old, male or female
• Able to understand the rationale of the clinical trial and sign informed consent form (ICF)
• Diagnosis of Early-onset Parkinson's Disease in accordance with the MDS clinical diagnostic criteria for Parkinson's disease, and the age of onset was ≤50 years old
• Medically suitable for neurosurgery under anesthesia and able to participate in Computed Tomography (CT)/Magnetic Resonance Imaging (MRI) scan.
• Hoehn-Yahr staging for "off" episodes is 2 to 4
• The MDS-UPDRS-III score in the "off" state >30, and positive for the Acute Levodopa Challenge Test (ALCT)
• Acceptable laboratory test results during screening and prior to transplantation

Exclusion Criteria

• Atypical Parkinsonism
• Patients who have had previous pallidotomy, striatal or extrapyramidal surgery 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 with surgical contraindications or other neurosurgical contraindications
• 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 received stem cell therapy for Parkinson's disease within 2 years before signing the ICF
• 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
• 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 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

iRegene Therapeutics Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Tongji Hospital Affiliated to Tongji Medical College Hust

Wuhan, Hubei, China

Site Status: RECRUITING

Countries

China

Central Contacts

Meng Cai

Role: CONTACT

caimeng@iregene.com

86-027-59338180

Facility Contacts

Wei Wang

Role: primary

wwang@vip.126.com

027-83663337

Xiang Luo

Role: backup

flydottjh@163.com

027-83663337

Wei Wang

Role: backup

Xiang Luo

Role: backup

Huaqiu Zhang

Role: backup

Other Identifiers

NouvNeu001-03

Identifier Type: -

Identifier Source: org_study_id