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

iRegene Therapeutics

Status and phase

Enrolling

Phase 1

Conditions

Early-onset Parkinson's Disease

Treatments

Biological: Human Dopaminergic Progenitor Cells

Study type

Interventional

Funder types

Industry

Identifiers

NCT06608355

NouvNeu001-03

Details and patient eligibility

About

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.

Enrollment

6 estimated patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

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