The Safety and Efficacy of NouvSoma001 in Neuromyelitis Optica Spectrum Disorders

NCT06620809 | Recruiting Phase 1

• 1 other identifier: NouvSoma001inNMOSD
• Study Type: interventional
• Target: 69
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Neuromyelitis Optica Spectrum Disorders

Keywords

Neuromyelitis Optica Spectrum Disorders; extracelluar vesicles

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (23)

• The incidence and severity of all adverse events (AE) and serious adverse events (SAE)

  Up to 18 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.

  Up to 6 month after treatment initiation

Study Arms (2)

Extracellular vesicles group

EXPERIMENTAL

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 placebo group

PLACEBO COMPARATOR

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

Interventions

A placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injection DRUG

Extracellular vesicles placebo(5×10\^9 particles）

Also known as: NouvSoma001

Extracellular vesicles placebo group

Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection DRUG

Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection（5×10\^9 particles）

Also known as: NouvSoma001

Extracellular vesicles group

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

• 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)
• Any contraindications to lumbar puncture.
• Pregnant or breastfeeding women, and patients with plans to conceive during the trial.
• Patients with a known history of allergies to human-derived biological products or those with an allergic predisposition.
• Patients who have undergone hematopoietic stem cell transplantation or lymphatic irradiation before enrollment.
• Patients who have participated in any other clinical trial within the last 3 months.
• Patients with severe comorbidities, including immunodeficiency or coagulation disorders.
• Patients with active suicidal ideation within 6 months before screening or have a history of suicide attempts within 3 years before screening.
• Patients with severe psychiatric symptoms that prevent clinical cooperation.
• Patients with positive for alcohol addiction or drug abuse.
• Patients with malignant tumors.
• 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.
• Patients with persistent systemic infections and severe local infections.
• Patients unable to undergo magnetic resonance imaging during the trial.

Sponsors & Collaborators

• Tongji Hospital: lead
• iRegene Therapeutics Co., Ltd.: collaborator

Study Sites (1)

Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology

Wuhan, Hubei, 430000, China

RECRUITING

Related Publications (2)

• 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.

  PMID: 26807122 BACKGROUND

• 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.

  PMID: 38242900 BACKGROUND

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

Injections, Spinal

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Injections; Drug Administration Routes; Drug Therapy; Therapeutics

Study Officials

• Dai-shi Tian, MD

  Tongji Hospoital

  PRINCIPAL INVESTIGATOR

• Wei Wang, MD

  Tongji Hospoital

  PRINCIPAL INVESTIGATOR

• Chuan Qin, MD

  Tongji Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chuan Qin, MD

CONTACT

86-27-83663337; qinchuan712@126.com

Chuan Qin, MD

CONTACT

86-27-83663332; chuanqin@tjh.tjmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof.

Study Record Dates

• First Submitted: September 22, 2024
• First Posted: October 1, 2024
• Study Start: March 28, 2025
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): November 30, 2027
• Last Updated: January 21, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)