Actively Recruiting
A Phase I/III Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Putamen Transplantation of NouvNeu001 Injection for Multiple System Atrophy
Led by iRegene Therapeutics Co., Ltd. · Updated on 2026-04-14
9
Participants Needed
1
Research Sites
195 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the safety, tolerability, and preliminary effectiveness of a single injection of NouvNeu001, which contains human dopaminergic progenitor cells, in people with Multiple System Atrophy (MSA), specifically the Parkinsonian subtype. This Phase I/III clinical trial focuses on patients who have had MSA motor symptoms for no more than five years and whose current treatments do not fully control their symptoms. The study is sponsored by iRegene Therapeutics Co., Ltd. and aims to better understand the risks and potential benefits of this new treatment. Participants will be randomly assigned to one of two groups during the low-dose phase: one group will receive a single injection of NouvNeu001 into the putamen region of the brain, while the control group will not receive the injection. The trial is open-label with no masking. The study treatment is administered once, and participants will be followed for one year after the transplantation to assess outcomes. Throughout the 52 weeks after receiving the injection, participants will undergo safety monitoring for adverse events, using standard criteria. Effectiveness will be evaluated by changes in motor symptoms using the Unified Multiple System Atrophy Rating Scale (UMSARS) and Clinical Global Impression-Severity (CGI-S) scores at weeks 13, 26, and 52. Brain imaging with DAT PET will also be performed at week 52 to assess dopaminergic metabolism. Participants' overall health and ability to walk will be assessed, and adherence to the trial procedures will be closely monitored.
CONDITIONS
Brief Title
A Phase I/III Clinical Study to Evaluate NouvNeu001 Injection for Multiple System Atrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged between 30 and 70 years (inclusive), regardless of gender.
- Understands and agrees to comply with study procedures and provides written informed consent.
- Diagnosed with pathologically confirmed, clinically established, or clinically probable Multiple System Atrophy according to 2022 MDS criteria.
- Current treatments for core MSA symptoms are inadequately controlled.
- Duration of MSA-related motor symptoms is no more than 5 years.
- Ability to walk without human assistance, able to take at least 10 steps; use of assistive devices permitted.
- Life expectancy of at least 3 years.
- Agrees not to participate in other clinical studies for 24 months after administration of the investigational product.
You will not qualify if you...
- Neurological diseases 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 trials of new drugs or therapies.
- Use of medications in past 3 months affecting Parkinsonian symptoms, autonomic function, or safety evaluation.
- Clinically significant or unstable medical/surgical conditions that may affect safety or outcomes.
- History or ongoing treatment for recurrent stroke.
- Brain MRI showing significant pathological findings except small meningiomas or arachnoid cysts less than 1 cm.
- Advanced disease indicated by severe speech, swallowing, walking impairment, or frequent falls.
- History of substance or alcohol abuse within 12 months.
- Known allergy to investigational product or related drugs.
- Positive tests for active viral infections including HIV, Hepatitis B, or Hepatitis C.
- Severe liver, kidney, or heart insufficiency.
- Recent thrombocytopenia, bleeding disorders, or anticoagulant therapy (except low-dose aspirin).
- Pregnancy, lactation, or planning pregnancy.
- History of major psychiatric disorders or suicidal ideation.
- Contraindications for surgery or recent neurosurgical procedures.
- Clinically significant cardiac disease.
- Diagnosis of cancer.
- Family history of congenital or inherited immunodeficiency.
- Poor compliance or other diseases that may jeopardize safety or interfere with assessments.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 52 weeks post-transplant
Participants receive a single injection of Human Dopaminergic Progenitor Cells into the putamen/striatum region of the brain or no intervention if randomized to the control group.
Visits at Weeks 13, 26, and 52 post-transplant for assessments
Trial Site Locations
Total: 1 location
1
Beijing Tiantan Hospital, Capital Medical University
Beijing, Beijing Municipality, China, 100730
Actively Recruiting
Research Team
M
Meng Cai, Ph.D
J
Jing Zhao, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here