The Safety and Efficacy of NouvSoma001 in Neuromyelitis Optica Spectrum Disorders

An Open-Label Exploratory Clinical Trial to Assess the Safety and Efficacy of NouvSoma001 in the Treatment of Neuromyelitis Optica Spectrum Disorders

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 efficacy of intrathecal administration of human-induced neural stem cell-derived extracellular vesicles (NouvSoma001) for the treatment of neuromyelitis optica spectrum disorders.

Study Overview

• Status: Recruiting
• Conditions: Neuromyelitis Optica Spectrum Disorders
• Intervention / Treatment: Drug: Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection; Drug: A placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injection

Detailed Description

This is a single-center, randomized, open-label, placebo-controlled, dose-escalation trial. The study consists of two parts: Part 1 is a dose-escalation study, while Part 2 is 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. In Cohort 1, participants will receive 5 × 10^9 particles; in Cohort 2, they will receive 1.5 × 10^10 particles; and in Cohort 3, they will receive 3 × 10^10 particles. If no dose-limiting toxicities (DLTs) 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 of Science and Technology
      • Principal Investigator: Wei Wang, MD
      • Principal Investigator: Daishi Tian, MD
      • Contact: Chuan Qin, MD; Phone Number: 86-27-83663337
      • Contact: Chuan Qin, MD; Phone Number: 86-27-83663332
      • Principal Investigator: Chuan Qin, MD

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Patients must meet the 2015 International Consensus Diagnostic Criteria for Neuromyelitis Optica Spectrum Disorder (NMOSD) and test positive for AQP4 antibodies.
2. Symptom onset occurred within 7 days prior to enrollment, with associated severe pain, lower limb motor dysfunction, or urinary/bowel impairment.
3. Males or Females aged between 18 and 65 years.
4. The Expanded Disability Status Scale (EDSS) score prior to the current disease episode is ≤ 4.
5. Female participants of childbearing potential must present a negative pregnancy test at screening and agree to use effective contraception throughout the study period.
6. Informed consent must be obtained from the patient or their legal representative, with a signed consent form must be provided.

Exclusion Criteria:

1. Abnormal laboratory indicators of the subjects need to be excluded, including, but not limited to, the following indicators:

   White Blood Cell Count <3*10^9/L Neutrophil Count <1.5*10^9/L <1.5*10^9/L Hemoglobin <85 <85 g/L Platelet Count <80*10^9/L <80*10^9/L Serum Creatinine >1.5*ULN Total Bilirubin >1.5*ULN AST (GOT) >3*ULN ALT (GPT) >3*ULN Alkaline Phosphatase >2*ULN (AST = Aspartate Aminotransferase; GOT = Glutamic-Oxaloacetic Transaminase; ALT = Alanine Aminotransferase; GPT = Glutamic-Pyruvic Transaminase)

2. Any contraindications to lumbar puncture.
3. Pregnant or breastfeeding women, and patients with plans to conceive during the trial.
4. Patients with a known history of allergies to human-derived biological products or those with an allergic predisposition.
5. Patients who have undergone hematopoietic stem cell transplantation or lymphatic irradiation before enrollment.
6. Patients who have participated in any other clinical trial within the last 3 months.
7. Patients with severe comorbidities, including immunodeficiency or coagulation disorders.
8. Patients with active suicidal ideation within 6 months before screening or have a history of suicide attempts within 3 years before screening.
9. Patients with severe psychiatric symptoms that prevent clinical cooperation.
10. Patients with positive for alcohol addiction or drug abuse.
11. Patients with malignant tumors.
12. Patients who have experienced any of the following events within 12 weeks before enrollment: myocardial infarction, unstable ischemic heart disease, stroke, or New York Heart Association Class IV heart failure.
13. Patients with persistent systemic infections and severe local infections.
14. Patients unable to undergo magnetic resonance imaging during the trial.
15. Patients deemed 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 group will receive extracellular vesicles derived from human-induced neural stem cells for intrathecal injection once a day for 1 day.	Drug: Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection; Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection（5×10^9 particles）; Other Names: NouvSoma001
Placebo Comparator: Extracellular vesicles placebo group; Patients in this group will receive a placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injection once a day for 1 day.	Drug: A placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injection; Extracellular vesicles placebo(5×10^9 particles）; Other Names: NouvSoma001

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
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 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 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 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
Magnetic Resonance Imaging（MRI）scan of the brain and spinal cord at month 3、6		Up to 6 month after treatment initiation
The score of Visual analogue scale（VAS） at month 1、3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
Brief Pain Inventory - Short form (BPI-SF) at month 1、3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The score of Expanded Disability Status Scale （EDSS） at month 1、3、6 compared with baseline and control group.	The score of EDSS ranges from 0 to 10, and 10 represents the worst.	Up to 6 month after treatment initiation
The score of Fecal Incontinence Severity Index (FISI) at month 1、3、6 compared with baseline and control group.	The score of FISI ranges from 0 to 3, and 3 represents the worst.	Up to 6 month after treatment initiation
The score of Hauser Ambulance Index （contains The timed 25-foot walk）at month 1、3、6 compared with baseline and control group.	The score of HAI ranges from 1 to 11, and 11 represents the worst.	Up to 6 month after treatment initiation
The score of Hamilton Despression 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 value of Nfl、GFAP in the serum at month 1、3、6 compared with baseline and control group		Up to 6 month after treatment initiation
The value of white blood cell count in cerebrospinal fluid at month 3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The value of QAlb in cerebrospinal fluid at month 3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The value of Nfl in cerebrospinal fluid at month 3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The value of GFAP in cerebrospinal fluid at month 3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The value of IL-1β in cerebrospinal fluid at month 3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The value of IL-6 in cerebrospinal fluid at month 3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The value of TNF-α in cerebrospinal fluid at month 3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
The value of sTREM2 in cerebrospinal fluid at month 3、6 compared with baseline and control group.		Up to 6 month after treatment initiation
Examination of visual function at month 1、3、6	Orbital MRI and VEP (Visual evoked potential)	Up to 6 month after treatment initiation
Optical coherence tomography (OCT) at month 3、6		Up to 6 month after treatment initiation

Collaborators and Investigators

• Sponsor: Tongji Hospital
• Collaborators: iRegene Therapeutics Co., Ltd.
• Investigators: Principal Investigator: Dai-shi Tian, MD, Tongji Hospoital; Principal Investigator: Wei Wang, MD, Tongji Hospoital; Principal Investigator: Chuan Qin, MD, Tongji Hospital

Publications and helpful links

General Publications

• Dulamea AO, Sirbu-Boeti MP, Bleotu C, Dragu D, Moldovan L, Lupescu I, Comi G. Autologous mesenchymal stem cells applied on the pressure ulcers had produced a surprising outcome in a severe case of neuromyelitis optica. Neural Regen Res. 2015 Nov;10(11):1841-5. doi: 10.4103/1673-5374.165325.
• Xu H, Jiang W, Li X, Jiang J, Afridi SK, Deng L, Li R, Luo E, Zhang Z, Huang YA, Cui Y, So KF, Chen H, Qiu W, Tang C. hUC-MSCs-derived MFGE8 ameliorates locomotor dysfunction via inhibition of ITGB3/ NF-kappaB signaling in an NMO mouse model. NPJ Regen Med. 2024 Jan 20;9(1):4. doi: 10.1038/s41536-024-00349-z.

Study record dates

Study Major Dates

• Study Start (Actual): March 28, 2025
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): November 30, 2027

Study Registration Dates

• First Submitted: September 22, 2024
• First Submitted That Met QC Criteria: September 27, 2024
• First Posted (Actual): October 1, 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: Neuromyelitis Optica Spectrum Disorders; extracelluar vesicles
• Additional Relevant MeSH Terms: Nervous System Diseases; Autoimmune Diseases; Immune System Diseases; Eye Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Neuromyelitis Optica; Therapeutics; Drug Administration Routes; Drug Therapy; Injections; Injections, Spinal
• Other Study ID Numbers: NouvSoma001inNMOSD

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