This Clinical Trial is Designed to Evaluate the Safety, Tolerability, Feasibility and Preliminary Efficacy of NouvNeu001 (Human Dopaminergic Progenitor Cells Injection) in Patients With Parkinson's Disease.

NCT07102342 | Recruiting Phase 1 FDA Drug

• 1 other identifier: NouvNeu001-01
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase 1 clinical study to evaluate the safety, tolerability, feasibility, and preliminary efficacy of NouvNeu001 in patients with advanced Parkinson's Disease.

Conditions

Parkinson Disease

Keywords

NouvNeu001; Human Dopaminergic Progenitor Cells Injection; phase 1 study; Dopaminergic Progenitor Cells

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability

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

  24 weeks and 48 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 15 years post-transplant

Study Arms (1)

NouvNeu001

EXPERIMENTAL

Single injection of Human Dopaminergic Progenitor Cells into the bilateral putamen/striatum regions of the brain.

Biological: Human Dopaminergic Progenitor Cells

Interventions

Human Dopaminergic Progenitor Cells BIOLOGICAL

Single injection of Human Dopaminergic Progenitor Cells into the biliteral putamen/striatum regions of the brain

Also known as: NouvNeu001

NouvNeu001

Eligibility Criteria

• Age: 30 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age
• Male or female patient must be 30 to 75 years of age inclusive, at the time of signing the informed consent form (ICF).
• Type of Patient and Disease Characteristics
• Diagnosis of PD between past 4 to 20 years (meet MDS 2015 clinical diagnostic criteria for PD).
• H-Y staging (Appendix III) for "OFF" episodes is 2.5-4.0.
• MDS-UPDRS-III score \> 35, and positive for the Acute levodopa challenge test (ALCT) (improvement \> 30% for MDS UPDRS-III staging from OFF episodes to ON episodes) over two screening period visits. Baseline scores will be computed as the means of two screening period visits.
• No significant change in UPDRS-III scores between the two visits during the screening period.
• Patients who take stable doing of dopamine drug for at least 4 weeks prior to receiving the study drug.
• No significant change in dose and dosing schedule of the prescribed medicines for PD during the screening period.
• Medically suitable for neurosurgery under general anesthesia.
• Acceptable laboratory test values during screening and prior to transplantation (Day 0):
• Absolute neutrophil count ≥ 2.0 × 109/L
• White blood cell count ≥ 4.0 × 109/L
• Platelet count ≥ 100 × 109/L
• Aspartate aminotransferase (AST) and alanine transaminase (ALT) ≤ 2.5 x upper limit of normal (ULN)

You may not qualify if:

• Medical Conditions
• Atypical Parkinsonism (Parkinsonism-Plus syndrome, secondary parkinsonism, familial parkinsonism) Prior/Concomitant Therapy
• Patients who have had previous pallidotomy, DBS surgery, striatal or extrapyramidal surgery, brain stereotaxy, prior surgical or radiation therapy to the brain or spinal cord, or other brain surgery; as well as other surgical procedures that, in the investigator's judgment, could interfere with 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 who have had previous cellular therapy.
• Patients who have used glucocorticoids for an extended period (≥14 days) and at high doses (equivalent to prednisone ≥ 20 mg/day or other glucocorticoids at equivalent doses) within 3 months prior to signing the ICF. (excluding topical treatment)
• Patients who have used immunosuppressive drugs for an extended period (≥14 days) within 3 months prior to signing the ICF.
• Patients who have used antipsychotics, such as antidepressants, antimanic drugs, etc. within 3 months prior to signing the ICF and are deemed by the investigator to potentially impact the study assessment.
• Patients who have used botulinum toxin or other drugs for dystonia or muscle spasticity within 6 months prior to signing the ICF and are deemed by the investigator to potentially impact the study assessment.
• Prior/Concurrent Clinical Study Experience
• Patients who are participating in other clinical trials, or have been enrolled in other clinical studies within 3 months prior to the screening
• Patients with poor compliance based on clinical evaluation of the investigator. Diagnostic Assessments
• Patients with a history of dementia or a severe cognitive disorder; or those with obvious dementia or congnitive impairment detected during screening; MDS-UPDRS congnitive impairment score (section 1.1) \> 3 point at screening; or patinets with poor compliance, inability to accurately keep diary, and/or inability to sign ICF due to dementia.
• Severe depression as defined by HAMD ≥ 24 at screening.
• Severe anxiety as defined by HAMA ≥ 29 at screening.

Sponsors & Collaborators

• iRegene Therapeutics Co., Ltd.: lead

Study Sites (1)

Cornell University Weill Medical College

New York, New York, 10065, United States

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

Jing Zhao, PhD

CONTACT

zhaojing@iregene.com

Study Design

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

Study Record Dates

• First Submitted: July 28, 2025
• First Posted: August 3, 2025
• Study Start: January 12, 2026
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): September 1, 2032
• Last Updated: March 13, 2026

Record last verified: 2026-03

Locations

US (1)