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

iRegene Therapeutics

Status and phase

Enrolling

Phase 2

Phase 1

Conditions

Parkinson Disease

Treatments

Biological: Human Dopaminergic Progenitor Cells

Study type

Interventional

Funder types

Industry

Identifiers

NCT06167681

NouvNeu001-02

Details and patient eligibility

About

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.

Full description

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.

Enrollment

40 estimated patients

Sex

All

Ages

50 to 75 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

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