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

NCT06608355 | Recruiting Phase 1 FDA Drug

• 1 other identifier: NouvNeu001-03
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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

Keywords

Early-onset Parkinson's Disease; Dopaminergic Progenitor Cells

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability

  Incidence and severity of adverse events (AEs) and serious adverse events (SAEs).

  Enrollment to 96 weeks post-transplant.

Secondary Outcomes (1)

• The Motor Function and Non-motor Function

  Baseline to 48 weeks and 96 weeks post-transplant

Other Outcomes (2)

• Positron Emission Tomography Imaging

  Baseline to 48 weeks post-transplant.

• Dopamine Metabolism

  Baseline to 48 weeks post-transplant.

Study Arms (1)

NouvNeu001

EXPERIMENTAL

Biological: Human Dopaminergic Progenitor Cells

Interventions

Human Dopaminergic Progenitor Cells BIOLOGICAL

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

NouvNeu001

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• iRegene Therapeutics Co., Ltd.: lead

Study Sites (1)

Tongji Hospital Affiliated to Tongji Medical College Hust

Wuhan, Hubei, 430030, China

RECRUITING

MeSH Terms

Conditions

Parkinsonian Disorders

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders

Central Study Contacts

Meng Cai

CONTACT

86-027-59338180; caimeng@iregene.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 14, 2024
• First Posted: September 23, 2024
• Study Start: August 23, 2024
• Primary Completion: April 1, 2026
• Study Completion (Estimated): March 1, 2027
• Last Updated: November 7, 2024

Record last verified: 2024-09

Locations

CN (1)