The Safety and Efficacy of NouvSoma001 in Ischemic Stroke

An Open-label Exploratory Clinical Trial to Assess the Safety and Efficacy of NouvSoma001 in the Treatment of Ischemic Stroke

This is a single-center, randomized, open-label, placebo-controlled, dose-escalation trial. The objective of this research is to evaluate the safety, tolerability, and preliminary efficacy of intravenous administration of human-induced neural stem cell-derived extracellular vesicles (NouvSoma001) in the treatment of ischemic stroke.

Study Overview

• Status: Recruiting
• Conditions: Ischemic Stroke
• Intervention / Treatment: Drug: extracellular vesicles derived from human induced neural stem cell for intravenous injection; Drug: a placebo of extracellular vesicles derived from human induced neural stem cell for intravenous injection

Detailed Description

This is a single-center, randomized, open-label, placebo-controlled, dose-escalation trial. This study will consist of 2 parts, with Part 1 being a dose-escalation study and Part 2 being a dose-extension study based on the results of Part 1. Part 1 will follow a traditional 3+3 dose-escalation design, enrolling a total of 9 subjects. Cohort 1: receive 4×10^9 particles/kg, Cohort 2: receive 8×10^9 particles/kg, and Cohort 3: receive 1.6×10^10 particles/kg. If no dose-limiting toxicities (DLT) are observed within 2 weeks after the initial administration, a new cohort will be enrolled at the next higher dose level. If DLTs are observed in 1 participant, another 2 participants will be treated at the same dose level. Dose escalation will cease if DLTs are observed in more than 33% of the participants. In Part 2, the remaining 60 participants will be randomized in a 2:1 ratio to the treatment and placebo groups, with the dose level determined by the Data Safety Monitoring Board based on the results of Part 1.

• Study Type: Interventional
• Enrollment (Estimated): 69
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Chuan Qin, MD; Phone Number: 86-27-83663337; Email: qinchuan712@126.com
• Study Contact Backup: Name: Chuan Qin, MD; Phone Number: 86-27-83663332; Email: chuanqin@tjh.tjmu.edu.cn

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430000
      • Recruiting
      • Tongji Hospital affiliated to Tongji Medical College of Huazhong University ofScience and Technology
      • Principal Investigator: Wei Wang, MD
      • Contact: Chuan Qiun; Phone Number: 86-27-83663337; Email: qinchuan712@126.com
      • Contact: Chuan Qin; Phone Number: 86-27-83663332; Email: chuanqin@tjh.tjmu.edu.cn
      • Principal Investigator: Daishi Tian, MD
      • Principal Investigator: Chuan Qin, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. The age of the recruiters ranged from 18 to 75 years.
2. Clinical diagnosis of ischemic stroke, the time of stroke onset is known, and the onset occurred no more than 7 days before enrollment.
3. Magnetic resonance imaging (MRI) or computed tomography (CT) findings consistent with ischemic stroke.
4. At the time of enrollment, the NIHSS score is between 6 and 20; additionally, there is at least one limb with muscle strength ≤ Grade 3.
5. Patients who have the mental capacity to understand and participate in the study.
6. Informed consent was obtained from patients or their legal representatives.

Exclusion Criteria:

1. CT indicates intracranial hemorrhage, including hemorrhagic stroke, epidural hematoma, subdural hematoma, intraventricular hemorrhage, subarachnoid hemorrhage, or hemorrhagic transformation.
2. Patients with Alzheimer's disease, Parkinson's syndrome, or other neurodegenerative disorders.
3. Evidence of brain tumors, epilepsy, or a history of traumatic brain injury.
4. Presence of non-vascular diseases causing white matter lesions, such as carbon monoxide poisoning, multiple sclerosis, or adrenoleukodystrophy.
5. Rapid spontaneous neurological improvement during the screening period, defined as a reduction of NIHSS score by ≥ 8 points from symptom onset to the first administration.
6. Persistent systemic infection, severe local infection, or ongoing use of immunosuppressants.
7. Patients with malignant diseases or an expected survival of less than 5 years.
8. Significant hearing or vision impairments, language disorders, or claustrophobia that would hinder cooperation with neuropsychological assessments and MRI examinations.
9. Contraindications to MRI.
10. Patients unable to comply with follow-up requirements during the study.
11. Severe liver, renal, cardiac, or pulmonary insufficiency, hematologic disorders, or malignant tumors (Liver insufficiency is defined as ALT or AST levels greater than 1.5 times the upper normal limit; renal insufficiency is defined as serum creatinine levels greater than 1.5 times the upper normal limit).
12. Patients with alcohol addiction or those testing positive for drug abuse.
13. Patients with a history of severe allergies or known allergy to human biological products.
14. Pregnant or breastfeeding women, and those planning to conceive during the trial period.
15. Participation in other clinical trials within the past 3 months.
16. Patients considered unsuitable for participation by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: extracellular vesicles group; Patients in this arm will be given extracellular vesicles derived from human-induced neural stem cells for intravenous injection once a day for 7 days.	Drug: extracellular vesicles derived from human induced neural stem cell for intravenous injection; extracellular vesicles derived from human induced neural stem cell for intravenous injection（4×10^9 particles/kg）; Other Names: NouvSoma001
Placebo Comparator: extracellular vesicles placebo group; Patients in this arm will be given a placebo of extracellular vesicles derived from human-induced neural stem cells for intravenous injection once a day for 7 days.	Drug: a placebo of extracellular vesicles derived from human induced neural stem cell for intravenous injection; extracellular vesicles placebo (4×10^9 particles/kg); Other Names: NouvSoma001placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The incidence and severity of all adverse events (AE) and serious adverse events (SAE)	The assessment of adverse events and serious adverse events	Up to 6 month after treatment initiation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The incidence and severity of all adverse events (AE) and serious adverse events (SAE)	The assessment of adverse events and serious adverse events	Up to 18 month after treatment initiation
Magnetic Resonance Imaging(MRI) at month 3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The National Institutes of Health Stroke Scale, NIHSS	To assess NIHSS of subjects at month 1、3、6 after treatment initiation	Up to 6 month after treatment initiation
The Modified Rankin Scale (mRS)	To assess mRS of subjects within month 1、3、6 after treatment initiation	Up to 6 month after treatment initiation
The score of Mini-mental State Examination (MMSE) at month 1、3、6 compared with baseline and control group.	The score of MMSE ranges from 0 to 30, and 30 represents the best.	Up to 6 month after treatment initiation
The score of Montreal cognitive assessment scale (MoCA) at month 1、3、6 compared with baseline and control group.	The score of MoCA ranges from 0 to 30, and 30 represents the best.	Up to 6 month after treatment initiation
The score of Barthel Index for activities of daily living (ADL) at month 1、3、6 compared with baseline and control group.	The score of ADL ranges from 0 to 100, and 100 represents the best.	Up to 6 month after treatment initiation
The score of Hamilton Depression Scale at month 1、3、6 compared with baseline and control group.	The score of Hamilton Depression Scale ranges from 0 to 81, and 81 represents the worst.	Up to 6 month after treatment initiation
The score of Hamilton Anxiety Scale at month 1、3、6 compared with baseline and control group.	The score of Hamilton Anxiety Scale ranges from 0 to 56, and 56 represents the worst.	Up to 6 month after treatment initiation
The score of Clock Drawing Test at month 1、3、6 compared with baseline and control group		Up to 6 month after treatment initiation
The value of Quality of Life (EQ-5D-5L) at month 1、3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The score of Alzheimer's disease assessment scale-cognitive section (ADAS-cog) at month 1、3、6 compared with baseline and control group.	The score of Alzheimer's disease assessment scale-cognitive section ranges from 0 to 70, and 70 represents the worst.	Up to 6 month after treatment initiation
The score of Clinical Dementia Rating (CDR) at month 1、3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The score of Hopkins verbal learning test（HVLT）at month 1、3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The incidence of Columbia-Suicide Severity Rating Scale (C-SSRS) events at month 1、3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The value of Nfl and GFAP in serum at month 1、3、6 compared with baseline and control group.		Up to 6 month after treatment initiation

Collaborators and Investigators

• Sponsor: Wei Wang
• Collaborators: iRegene Therapeutics Co., Ltd.
• Investigators: Principal Investigator: Wei Wang, MD, Tongji hospital; Principal Investigator: Daishi Tian, MD, Tongji hospital; Principal Investigator: Chuan Qin, MD, Tongji hospital

Publications and helpful links

General Publications

• Zhang R, Mao W, Niu L, Bao W, Wang Y, Wang Y, Zhu Y, Yang Z, Chen J, Dong J, Cai M, Yuan Z, Song H, Li G, Zhang M, Xiong N, Wei J, Dong Z. NSC-derived exosomes enhance therapeutic effects of NSC transplantation on cerebral ischemia in mice. Elife. 2023 Apr 27;12:e84493. doi: 10.7554/eLife.84493.
• Zhu ZH, Jia F, Ahmed W, Zhang GL, Wang H, Lin CQ, Chen WH, Chen LK. Neural stem cell-derived exosome as a nano-sized carrier for BDNF delivery to a rat model of ischemic stroke. Neural Regen Res. 2023 Feb;18(2):404-409. doi: 10.4103/1673-5374.346466.
• Gao G, Li C, Ma Y, Liang Z, Li Y, Li X, Fu S, Wang Y, Xia X, Zheng JC. Neural stem cell-derived extracellular vesicles mitigate Alzheimer's disease-like phenotypes in a preclinical mouse model. Signal Transduct Target Ther. 2023 Jun 14;8(1):228. doi: 10.1038/s41392-023-01436-1. No abstract available.

Study record dates

Study Major Dates

• Study Start (Actual): November 30, 2025
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): November 30, 2027

Study Registration Dates

• First Submitted: September 22, 2024
• First Submitted That Met QC Criteria: September 24, 2024
• First Posted (Actual): September 25, 2024

Study Record Updates

• Last Update Posted (Actual): January 21, 2026
• Last Update Submitted That Met QC Criteria: January 18, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: Ischemic Stroke; extracellular vesicles
• Additional Relevant MeSH Terms: Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Stroke; Ischemic Stroke; Therapeutics; Drug Administration Routes; Drug Therapy; Administration, Intravenous; Injections; Injections, Intravenous
• Other Study ID Numbers: NouvSoma001inIS

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No