A Phase 1 interventional study of Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection and A placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injection in Neuromyelitis Optica Spectrum Disorders, sponsored by Tongji Hospital. Recruiting at 1 site in China. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-09-21.
Sponsored by Tongji Hospital · Phase 1, Interventional, and Treatment
Among 72 studies in Neuromyelitis Optica with a site in China — 37 open to participants now, 43 under way, 7 of them phase 3. Neuromyelitis Optica · China.
Among the 72 studies in Neuromyelitis Optica with a site in China, this one is the 13th largest by planned enrollment of the 51 interventional ones that give a count, is one of 10 that started in 2025, is one of 17 in phase 1, reaches primary completion later than 29 of the 42 other live ones, and was the 49th registered.
178 studies on the registry are indexed under Neuromyelitis Optica; 78 are open to participants now.
Browse Neuromyelitis Optica studies →37 of the 72 studies in Neuromyelitis Optica with a site in China are open now. Recruiting first, the newest first:
Alexion Pharmaceuticals, Inc. · Sites in China
Junwei Hao, MD · Sites in China
Hoffmann-La Roche · Sites in China, United States, Argentina and 6 more countries
Tongji Hospital · Sites in China
Chinese PLA General Hospital · Sites in China
A study can be open while its sites in China have not opened yet or have stopped. Only those recruiting at a site in China →
Of the 50 Neuromyelitis Optica studies recruiting now, 29 (58%) set no upper age limit and 16 (32%) stop at 75 or younger; 11 take children under 18; 9 take healthy volunteers.
This study: 18 Years to 65 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
72 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 23 · Phase 2 17 · Phase 3 9 · Phase 4 6
First in 2009; this study’s year is marked. 10 started in 2025.
This study's planned enrollment of 69 is above the median of 29 across 108 interventional studies indexed under Neuromyelitis Optica.
Median enrollment 23 across the 51 interventional studies, 300 across the 21 observational. Of the 17 that completed or were terminated, 0 (0%) have results posted.
This tab as a report to print or save as PDF → With an account.
18 of 72 have an industry lead sponsor. The 10 most frequent:
Tongji Hospital is the lead sponsor of 386 studies on the registry; 209 are open to participants now.
Tongji Hospital has 7 studies filed under Neuromyelitis Optica — fewer than ten, so there is no comparison to draw.
19 of the studies in Neuromyelitis Optica with a site in China that are under way reach primary completion within a year, 4 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06724809Phase 3Alexion Pharmaceuticals, Inc.Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD | 2026-12-07 |
| NCT07420296Phase 3Tianjin Medical University General HospitalModified Zipper Therapy for AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder | 2026-12-31 |
| NCT05199688Phase 3Hoffmann-La RocheA Study to Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, and Pharmacodynamics of Satralizumab in Pediatric Patients With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD) | 2027-03-31 |
| NCT06413654Phase 3Shanghai Jiaolian Drug Research and Development Co., LtdA Clinical Study of B001 Injection in the Treatment of Neuromyelitis Optica Spectrum Disorders(NMOSD) | 2027-10-31 |
| NCT05145361Shanghai Pharmaceuticals Holding Co., LtdClinical Study of B001 Injection in Subjects With Neuromyelitis Optic Spectrum Disorder (NMOSD) | 2026-12-15 |
Among the 72 studies in Neuromyelitis Optica with a site in China, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
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.
The sample size of this study is not based on statistical hypothesis testing. A total of 69 participants are planned to be enrolled, including 9 participants in the dose-escalation study and 60 participants in the dose-expansion study.
Part 1 will adopt a traditional 3 + 3 dose-escalation design, with a planned enrollment of 9 participants. Participants will be assigned into three cohorts:
Cohort 1: 5 × 10⁹ particles Cohort 2: 1.5 × 10¹⁰ particles Cohort 3: 4.5 × 10¹⁰ particles
If no dose-limiting toxicities (DLTs) are observed within 2 weeks after treatment in the first participant enrolled in Cohort 1, two additional participants may be enrolled. If no DLTs are observed within 2 weeks after treatment in these two additional participants, and at least one of the three participants in the same dose cohort demonstrates a therapeutic response, investigators may make the following decisions based on the preliminary efficacy signals and safety data obtained:
If the first three participants enrolled in Cohort 1 do not experience any DLTs, but none of the participants demonstrate clinical efficacy, and investigators have no additional safety concerns regarding NouvSoma001 extracellular vesicle injection, dose escalation to Cohort 2 (1.5 × 10¹⁰ particles) will be performed for DLT evaluation.
If one participant among the first three participants in Cohort 1 experiences a DLT, enrollment in the 5 × 10⁹ particles dose cohort will continue until a total of 6 DLT-evaluable participants are included.
If two participants among the first three participants in Cohort 1 experience DLTs, investigators may decide, based on the preliminary efficacy and safety data obtained, whether to evaluate a lower dose level (2.5 × 10⁹ particles) for further safety and efficacy assessment.
Cohorts 2 and 3 will follow the same dose-escalation rules as Cohort 1 until completion of Part 1.
In Part 2, the remaining 60 participants will be randomly assigned in a 2:1 ratio to the treatment group and placebo group. The dose level used in Part 2 will be determined based on the results of Part 1.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Abnormal laboratory indicators of the subjects need to be excluded, including, but not limited to, the following indicators:
White blood cell count \< 3 × 10⁹/L \< 1.5 × 10⁹/L \< 85 g/L count \< 80 × 10⁹/L Serum creatinine > 1.5 × Total > 1.5 × ULN (AST) > 3 × ULN Alanine aminotransferase (ALT) > 3 × ULN Alkaline phosphatase > 2 × ULN (AST = Aspartate Aminotransferase; GOT = Glutamic-Oxaloacetic Transaminase; ALT = Alanine Aminotransferase; GPT = Glutamic-Pyruvic Transaminase)
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
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 derived from human-induced neural stem cells for intrathecal injection
Also known as: NouvSoma001
A placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injection
The incidence and severity of all adverse events (AE) and serious adverse events (SAE)
The assessment of adverse events and serious adverse events
Time frame: Up to 6 month after treatment initiation
The incidence and severity of all adverse events (AE) and serious adverse events (SAE)
The assessment of adverse events and serious adverse events
Time frame: Up to 18 month after treatment initiation
Magnetic Resonance Imaging (MRI) scans of the brain and spinal cord at month 1 and month 6
Time frame: Up to 6 month after treatment initiation
The score of Visual analogue scale（VAS）
Time frame: Up to 6 month after treatment initiation
Brief Pain Inventory - Short form (BPI-SF)
Time frame: Up to 6 month after treatment initiation
The score of Expanded Disability Status Scale （EDSS）
Time frame: Up to 6 month after treatment initiation
The score of Fecal Incontinence Severity Index (FISI)
Time frame: Up to 6 month after treatment initiation
The score of Hauser Ambulance Index （contains The timed 25-foot walk）
Time frame: Up to 6 month after treatment initiation
The score of Hamilton Anxiety Rating Scale
Time frame: Up to 6 month after treatment initiation
The score of Hamilton Depression Rating Scale
Time frame: Up to 6 month after treatment initiation
The Modified Rankin Scale (mRS)
Time frame: Up to 6 month after treatment initiation
The value of Quality of Life (EQ-5D-5L)
Time frame: Up to 6 month after treatment initiation
The incidence of Columbia-Suicide Severity Rating Scale (C-SSRS) events
Time frame: 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
Time frame: Up to 6 month after treatment initiation
The value of white blood cell count in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Time frame: Up to 6 month after treatment initiation
The value of QAlb in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Time frame: Up to 6 month after treatment initiation
The value of Nfl in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Time frame: Up to 6 month after treatment initiation
The value of GFAP in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Time frame: Up to 6 month after treatment initiation
The value of IL-1β in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Time frame: Up to 6 month after treatment initiation
The value of IL-6 in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Time frame: Up to 6 month after treatment initiation
The value of TNF-α in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Time frame: Up to 6 month after treatment initiation
The value of sTREM2 in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Time frame: Up to 6 month after treatment initiation
Examination of visual function
Time frame: Up to 6 month after treatment initiation
Optical coherence tomography (OCT)
Time frame: Up to 6 month after treatment initiation
Translocator Protein (TSPO) PET imaging at month 1
Time frame: Up to 1 month after treatment initiation
Chest computed tomography (CT) scans at month 6 and month 18
Time frame: Up to 18 month after treatment initiation
Tumor marker assessment at month 6
Time frame: Up to 6 month after treatment initiation
Plan to share: No
Started Mar 2025 (actual). Primary completion is estimated for Nov 2027, 32 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.