Home NouvNeu001 Intrastriatal Transplantation for Multiple System Atrophy-Parkinsonian Type (MSA-P): A Phase I/III Clinical Trial

NouvNeu001 Intrastriatal Transplantation for Multiple System Atrophy-Parkinsonian Type (MSA-P): A Phase I/III Clinical Trial

Dec 29, 2025 20:30 CST Updated 20:30
iRegene Therapeutics

Cell Therapy Product Developer

Multiple System Atrophy Parkinsonian Type (MSA-P) is a rare and rapidly progressing adult-onset neurodegenerative disease, belonging to the synucleinopathies. Currently, its clinical treatment faces severe challenges. Existing drugs such as levodopa can provide temporary relief of some motor symptoms in certain patients, but their overall efficacy is limited and they cannot slow disease progression. More problematic is that the disease is often accompanied by severe autonomic dysfunction (such as orthostatic hypotension, urinary dysfunction, etc.) and cerebellar ataxia, which are poorly responsive to drug treatments, leading to a sharp decline in patients' quality of life and very poor prognosis. Therefore, exploring disease-modifying therapies that can fundamentally slow or halt disease progression and improve core functional impairments is the most urgent need in current clinical research and among patient populations.

Human-Derived Dopaminergic Precursor Cells (NouvNeu001)InjectionAs a novel cell therapy product, it provides a new direction to break through this dilemma. By injecting into the putamen of the brain to replenish damaged dopaminergic neurons, it is expected to rebuild neural circuit function, reverse pathological damage, and achieve disease-modifying treatment for MSA-P, bringing new hope to patients with poor symptom control.

To scientifically evaluate the clinical value of this innovative cell therapy product, a clinical trial was initiated by Professor Wang Yilong's team from Beijing Tiantan Hospital, Capital Medical University, focusing on the assessment of NouvNeu001.InjectionThe safety and tolerability, aiming to provide the first precision-targeted cell therapy solution for MSA-P, to help improve patients' quality of life and prognosis.

Reference: "Chinese Expert Consensus on the Diagnostic Criteria for Multiple System Atrophy (2022)"




Introduction to the Study

Indications:Multiple System Atrophy - Parkinsonian Type (MSA-P)

Therapeutic Drugs:Human-derived dopaminergic precursor cells (NouvNeu001)Injection

Research Purpose:Evaluate the safety and tolerability of intraputamenal injection of NouvNeu001 for the treatment of Parkinsonian variant of Multiple System Atrophy (MSA-P), and determine the Maximum Tolerated Dose (MTD) or the recommended dose for Phase 2.

Study Design:Open, Parallel Group, Randomization

Research Phase:Phase I/III

This study (CTR20254863) was sponsored by iRegene (Chengdu) and iRegene (Wuhan).Professor Wang Yilong's Team from Beijing Tiantan Hospital, Capital Medical University(PI) initiated, and has currently been approved by the Medical Ethics Committee of Beijing Tiantan Hospital, Capital Medical University.



Participating Institutions



  • Beijing Tiantan Hospital, Capital Medical University - Beijing


Inclusion Criteria

1.30 years old ≤ age ≤ 70 years old, gender不限

2. The subject understands and complies with the study procedures, voluntarily participates, and signs the informed consent form.

3. Diagnosed as pathologically confirmed, clinically confirmed, or clinically probable MSA according to the diagnostic criteria proposed in the 2022 version of MDS.

4. Subjects with poor control of core MSA symptoms currently

5. The time since the onset of MSA-related motor symptoms (parkinsonism and/or cerebellar ataxia) in the subject does not exceed 5 years.

6. Can walk independently without assistance from others, defined as being able to walk at least 10 steps, with the allowance of using assistive devices (e.g., walkers or canes).

7. Expected survival time is at least 3 years

8. The subject agrees not to participate in any other clinical studies within 24 months after dosing.




Exclusion Criteria

1.Neurological diseases/disorders other than multiple system atrophy, such as Parkinson's disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, spinocerebellar ataxia, spastic paraplegia, corticobasal degeneration, vascular parkinsonism, normal pressure hydrocephalus, drug-induced or post-encephalitic parkinsonism.

2. The subject was diagnosed with dementia.

3. Have previously or are currently receiving other disease-modifying treatments, or participating in clinical studies of other new drugs or therapies.

4. Subjects who have used drugs that may affect Parkinson's symptoms, potentially impact autonomic nervous function, or influence safety evaluation within the past 3 months.

5. Subjects with clinically significant or unstable medical or surgical conditions that may prevent safe completion of treatment or affect treatment outcomes.

6. Recurrent stroke that has been previously suffered from or is currently under treatment

7. Brain magnetic resonance imaging at the time of screening showed other significant pathological findings, including but not limited to: cerebral hemorrhage, acute phase cerebral infarction, aneurysm, vascular malformation, infectious lesions, brain tumors, or other space-occupying lesions (meningiomas or arachnoid cysts with a maximum diameter of less than 1 cm do not need to be excluded).

8. Subjects meeting any of the following criteria indicating a more severe condition: Speech impairment determined by a score of ≥3 on Question 1 of the UMSARS questionnaire; Swallowing impairment determined by a score of ≥3 on Question 2 of the UMSARS questionnaire; Gait impairment determined by a score of ≥3 on Question 7 of the UMSARS questionnaire; Falling more than once per week determined by a score of ≥3 on Question 8 of the UMSARS questionnaire.

9. The subject has a history of current drug and/or alcohol abuse (within 12 months prior to trial enrollment).

10. The subject is known to be allergic to the investigational drug; or has a history of allergies to drugs such as antibiotics.

11. Screening results show active viral infections, including positivity for Human Immunodeficiency Virus (HIV), Hepatitis B Surface Antigen (HBsAg), Hepatitis B Core Antibody, and Hepatitis C Virus (HCV).

12. Accompanied by severe hepatic insufficiency, renal insufficiency, or severe cardiac insufficiency (Severe hepatic insufficiency refers to alanine aminotransferase (ALT) values ≥ 2.0 times the upper limit of normal or aspartate aminotransferase (AST) values ≥ 2.0 times the upper limit of normal; Severe renal insufficiency refers to serum creatinine ≥ 1.5 times the upper limit of normal or estimated glomerular filtration rate (eGFR) < 40 ml/min/1.73 m²; Severe cardiac insufficiency refers to New York Heart Association (NYHA) functional classification grade 3-4).

13. Subjects with thrombocytopenia within three months, subjects with other bleeding disorders or receiving anticoagulation therapy (excluding aspirin not exceeding 100 mg per day)

14. Subjects who are pregnant, lactating, or possibly pregnant and planning to become pregnant

15. History of bipolar disorder, major depressive disorder, schizophrenia, or other psychotic disorders

16. Subjects who are clinically judged to have suicidal ideation at the time of screening, or who have attempted suicide within 6 months prior to screening.

17. Those with surgical contraindications (such as having previously received a cochlear implant, pacemaker, defibrillator, stereotactic neuroablation, or having previously been implanted with similar products unilaterally or bilaterally, etc.), or those who have undergone other surgeries within the past six months that the investigator believes may impact this trial, or those with other neurosurgical contraindications.

18. Clinically significant heart disease-related indicators are defined as: myocardial infarction, heart failure with NYHA class III or IV, uncontrolled coronary artery spasm, severe and uncontrolled ventricular arrhythmia, or evidence of acute ischemia or abnormal conduction system on electrocardiogram within 6 months prior to enrollment.

19. Subjects with malignant tumors

20. Family history of congenital or hereditary immunodeficiency diseases

21. Participants assessed by the investigator as having poor compliance

22. Suffering from a serious illness, or having any other condition that endangers the safety of the subject or affects the investigator's assessment.



Research Benefits

Purchase insurance during the trial period to provide security for patients.

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Introduction of the Principal Investigator

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Professor Wang Yilong

Beijing Tiantan Hospital, Capital Medical University

  • Chief Physician, Professor, Doctoral Supervisor, Beijing Scholar.

  • Currently serving as the Executive Vice Dean of Beijing Tiantan Hospital, Capital Medical University, Deputy Director of the National Center for Neurological Disorders, and Director of the Tiantan Neurology Rare Diseases Clinical and Translational Center.

  • Awarded the National Youth Science Foundation (Category A) and the first batch of continuation funding, and selected as a leading talent in national science and technology innovation, etc.

  • Long-term dedication to clinical and translational research on cerebral small vessel disease and rare neurological disorders: Pioneering a new theory and technology for drug delivery through the transcranial bone marrow-dura mater-glymphatic system, bypassing the blood-brain barrier, based on the cranial-brain immune-metabolic mechanism. This breakthrough overcomes conventional drug delivery methods, enabling rapid and efficient drug administration to the central nervous system; completed First-In-Man (FIM) clinical validation with good safety and efficacy; research findings have been accepted by journals such as The Lancet and Cell.

  • Developed the world's first transcranial bone marrow drug delivery device, receiving clinical trial approval from the National Medical Products Administration (NMPA).

  • Establish precise intensified antithrombotic and balloon-first endovascular treatment techniques, significantly reducing the risk of stroke recurrence, incorporated into cerebrovascular disease guidelines.

  • The research findings were published in top journals such as The New England Journal of Medicine (N Engl J Med), The Journal of the American Medical Association (JAMA), and The British Medical Journal (BMJ); developed two Class 2 new drugs, one Class 1.1 new drug, and two Class 3 medical devices.

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