Safety and effects of NouvNeu001 for early-onset Parkinson's Disease

A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of NouvNeu001 Injection in the Treatment of Early-onset Parkinson's Disease

This study tests if NouvNeu001 injections can safely improve symptoms for people with early-onset Parkinson's Disease.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: iRegene Therapeutics Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Wuhan, Hubei)
• Trial ID: NCT06608355 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the safety and tolerability of NouvNeu001 injections in patients with early-onset Parkinson's Disease (EOPD). Participants will receive the treatment via stereotactic neurosurgery, and the study will assess both motor and non-motor function improvements post-injection. Additionally, participants will take immunosuppressants for 24 to 36 weeks to prevent immune rejection of the transplanted cells. The trial will focus on understanding the medical issues related to the procedure and the overall efficacy of the treatment.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 18-70 diagnosed with early-onset Parkinson's Disease, with an onset age of 50 or younger.

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

Why it matters

Potential benefit: If successful, this treatment could significantly improve motor and non-motor functions in patients with early-onset Parkinson's Disease.

How similar studies have performed: While this approach is novel, similar studies involving dopaminergic progenitor cells have shown promise in other contexts.

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

Where this trial is running

Wuhan, Hubei

• Tongji Hospital Affiliated to Tongji Medical College Hust — Wuhan, Hubei, China (Recruiting)

Study contacts

• Study coordinator: Meng Cai

Conditions Early-onset Parkinson's Disease; Dopaminergic Progenitor Cells