A Phase I/III Clinical Study to Evaluate NouvNeu001 Injection for Multiple System Atrophy

NCT07289477 | Recruiting Phase 1 FDA Drug

• 1 other identifier: NouvNeu004-01
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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 Multiple System Atrophy.

Conditions

Multiple System Atrophy - Parkinsonian Subtype (MSA-P)

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of Adverse Events as Assessed by CTCAE V5.0

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

  52 weeks post-transplant

Secondary Outcomes (5)

• Change from baseline in the UMSARS total score (Part I + Part II) at Weeks 13, 26, and 52.

  Weeks 13, 26, and 52 post-transplant

• Change from baseline in the UMSARS Part I score at Weeks 13, 26, and 52.

  Weeks 13, 26, and 52 post-transplant

• Change from baseline in the UMSARS Part II score at Weeks 13, 26, and 52.

  Weeks 13, 26, and 52 post-transplant

• Change from baseline in dopaminergic metabolism of the basal ganglia at Week 52, as measured by DAT PET imaging.

  Weeks 52 post-transplant

• Change from baseline in the CGI-S score at Weeks 13, 26, and 52.

  Weeks 13, 26, and 52 post-transplant

Other Outcomes (2)

• Pharmacodynamic Biomarkers (NfL)

  Weeks 13, 26, and 52 post-transplant

• Pharmacodynamic Biomarkers (α-syn)

  Weeks 13, 26, and 52 post-transplant

Study Arms (2)

NouvNeu001

EXPERIMENTAL

During the enrollment phase for the low-dose cohort, 3 participants will be randomized to the experimental arm to receive a single administration of NouvNeu001 Injection.

Biological: Human Dopaminergic Progenitor Cells

control

NO INTERVENTION

During the enrollment phase for the low-dose cohort, 3 participants will be randomized to the control arm, which will not receive NouvNeu001 Injection.

Interventions

Human Dopaminergic Progenitor Cells BIOLOGICAL

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

NouvNeu001

Eligibility Criteria

• Age: 30 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged between 30 and 70 years (inclusive), regardless of gender.
• The subject understands and agrees to comply with the study procedures and voluntarily provides written informed consent.
• Diagnosed with pathologically confirmed, clinically established, or clinically probable Multiple System Atrophy (MSA) according to the 2022 MDS diagnostic criteria.
• Current treatments for core MSA symptoms are inadequately controlled.
• The duration of MSA-related motor symptoms (parkinsonism and/or cerebellar ataxia) is no more than 5 years.
• Ability to walk without human assistance, defined as being able to take at least 10 steps; the use of assistive devices (e.g., a walker or cane) is permitted.
• Life expectancy of at least 3 years.
• The subject agrees not to participate in any other clinical studies for 24 months following the investigational product administration.

You may not qualify if:

• Neurological diseases/disorders other than Multiple System Atrophy, such as Parkinson's disease, Dementia with Lewy Bodies, Essential Tremor, Progressive Supranuclear Palsy, Spinocerebellar Ataxia, Hereditary Spastic Paraplegia, Corticobasal Degeneration, Vascular Parkinsonism, Normal Pressure Hydrocephalus, or Drug-induced/Postencephalitic Parkinsonism.
• Diagnosis of dementia.
• Previous or current receipt of other disease-modifying therapies, or participation in clinical trials of other new drugs or novel therapies.
• Use of medications within the past 3 months that may affect Parkinsonian symptoms, autonomic function, or the evaluation of safety.
• Presence of clinically significant or unstable medical or surgical conditions that may preclude the safe completion of the treatment or confound the treatment outcomes.
• History of or undergoing treatment for recurrent stroke.
• Subjects meeting any of the following criteria indicating advanced disease:
• Speech impairment defined by a score of ≥3 on UMSARS Item 1. Swallowing impairment defined by a score of ≥3 on UMSARS Item 2. Walking impairment defined by a score of ≥3 on UMSARS Item 7. Occurrence of falls more than once per week, defined by a score of ≥3 on UMSARS Item 8.
• History of current substance abuse and/or alcohol abuse (within 12 months prior to screening).
• Known allergy to the investigational product(s); or history of allergy to antibiotics or other drugs.
• Positive screening results for active viral infection, including Human Immunodeficiency Virus (HIV), Hepatitis B Surface Antigen (HBsAg), Hepatitis B Core Antibodies, or Hepatitis C Virus (HCV).
• Severe hepatic insufficiency, renal insufficiency, or severe cardiac insufficiency:
• Severe hepatic insufficiency: ALT ≥ 2.0 × upper limit of normal (ULN) or AST ≥ 2.0 × ULN.
• Severe renal insufficiency: Serum creatinine ≥ 1.5 × ULN or estimated Glomerular Filtration Rate (eGFR) \< 40 mL/min/1.73 m².
• Severe cardiac insufficiency: New York Heart Association (NYHA) Class 3 or 4.

Sponsors & Collaborators

• iRegene Therapeutics Co., Ltd.: lead

Study Sites (1)

Beijing Tiantan Hospital, Capital Medical University

Beijing, Beijing Municipality, 100730, China

RECRUITING

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: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 26, 2025
• First Posted: December 17, 2025
• Study Start: January 5, 2026
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): July 1, 2031
• Last Updated: April 14, 2026

Record last verified: 2025-11

Locations

CN (1)