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

NCT06167681 | Recruiting Phase 1 FDA Drug

• 1 other identifier: NouvNeu001-02
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Parkinson Disease

Keywords

Parkinson Disease; Dopaminergic Progenitor Cells

Outcome Measures

Primary Outcomes (2)

• Safety and Tolerability

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

  24 weeks and 48 weeks post-transplant

• Motor Function

  Changes in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part Ⅲ score from baseline to 24 weeks post-transplant. MDS-UPDRS Part III assesses the motor signs of PD, a higher score indicates more severe symptoms of PD.

  24 weeks post-transplant

Secondary Outcomes (2)

• The Motor Function and Non-motor Function

  48 weeks and 96 weeks post-transplant

• Continued Safety and Tolerability

  96 weeks and 5 years 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/striatum region of brain.

NouvNeu001

Eligibility Criteria

• Age: 50 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• iRegene Therapeutics Co., Ltd.: lead

Study Sites (2)

Beijing Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

Zhongnan Hospital of Wuhan University

Wuhan, Hubei, 430071, China

NOT YET RECRUITING

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Central Study Contacts

Meng Cai, Ph.D

CONTACT

0086-027-59337986; 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: November 24, 2023
• First Posted: December 12, 2023
• Study Start: January 17, 2024
• Primary Completion: December 1, 2025
• Study Completion (Estimated): July 1, 2029
• Last Updated: November 1, 2024

Record last verified: 2024-07

Locations

CN (2)