Evaluating a new treatment for Parkinson's disease using human progenitor cells

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

This study is testing a new cell treatment for Parkinson's disease to see if it is safe and helps improve symptoms in patients.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 50 Years to 75 Years
• Sex: All
• Sponsor: iRegene Therapeutics Co., Ltd. (Industry-sponsored)
• Locations: 2 sites (Beijing, Beijing Municipality and 1 other locations)
• Trial ID: NCT06167681 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety, tolerability, and preliminary efficacy of NouvNeu001, a human dopaminergic progenitor cell injection, in patients diagnosed with Parkinson's disease. It is a multi-center, single-arm, open-label trial where the treatment will be administered via stereotactic neurosurgery into the bilateral putamen/striatum. Participants will receive immunosuppressants for 24 to 36 weeks to prevent immune rejection of the transplanted cells.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 50-75 with a diagnosis of Parkinson's disease made between 4 to 20 years ago and who meet specific clinical criteria.

Not a fit: Patients with atypical Parkinsonism or those who have undergone certain previous brain surgeries will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a novel therapeutic option that may improve motor function and quality of life for patients with Parkinson's disease.

How similar studies have performed: While this approach is innovative, similar studies using cell-based therapies for Parkinson's disease have shown promise, indicating potential for success.

Eligibility criteria

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

Where this trial is running

Beijing, Beijing Municipality and 1 other locations

• Beijing Hospital — Beijing, Beijing Municipality, China (Recruiting)
• Zhongnan Hospital of Wuhan University — Wuhan, Hubei, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Meng Cai, Ph.D

Conditions Parkinson Disease; Dopaminergic Progenitor Cells