Neuromyelitis Optica Spectrum DisordersPhase 1NCT06620809
The Safety and Efficacy of NouvSoma001 in Neuromyelitis Optica Spectrum Disorders
A Phase 1 study testing Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection in people with Neuromyelitis Optica Spectrum Disorders, run by Tongji Hospital at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorders
- What is being tested?
- Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 69 participants
- Where
- 1 siteChina
- Study dates
- 28 Mar 2025to 30 Nov 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 Neutrophil count < 1.5 × 10⁹/L Hemoglobin < 85 g/L Platelet count < 80 × 10⁹/L Serum creatinine > 1.5 × ULN Total bilirubin > 1.5 × ULN Aspartate aminotransferase (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)
- 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.
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- 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.
- Patients deemed unsuitable for participation by the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology
Wuhan, Hubei, 430000
Chuan Qin, MD, 86-27-83663337
Chuan Qin, MD, 86-27-83663332
About this study
Official title: 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.
Read the detailed description
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:
- Continue enrollment in the 5 × 10⁹ particles dose cohort until a total of 6 DLT-evaluable participants are enrolled; or
- Stop further enrollment in the 5 × 10⁹ particles dose cohort and proceed to the next dose level (1.5 × 10¹⁰ particles) for DLT evaluation.
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.
- Conditions
- Neuromyelitis Optica Spectrum Disorders
- Being tested
- Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection, A placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injection
- Keywords
- Neuromyelitis Optica Spectrum Disorders, extracelluar vesicles
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Extracellular vesicles derived from human-induced neural stem cells for intrathecal injectionDrug
Also called NouvSoma001
Extracellular vesicles derived from human-induced neural stem cells for intrathecal injection
A placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injectionDrug
A placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injection
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: A placebo of extracellular vesicles derived from human-induced neural stem cells for intrathecal injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence and severity of all adverse events (AE) and serious adverse events (SAE)
The assessment of adverse events and serious adverse events
Measured: Up to 6 month after treatment initiation
Secondary outcomes (26)
The incidence and severity of all adverse events (AE) and serious adverse events (SAE)
The assessment of adverse events and serious adverse events
Measured: Up to 18 month after treatment initiation
Magnetic Resonance Imaging (MRI) scans of the brain and spinal cord at month 1 and month 6
Measured: Up to 6 month after treatment initiation
The score of Visual analogue scale（VAS）
Measured: Up to 6 month after treatment initiation
Brief Pain Inventory - Short form (BPI-SF)
Measured: Up to 6 month after treatment initiation
The score of Expanded Disability Status Scale （EDSS）
Measured: Up to 6 month after treatment initiation
The score of Fecal Incontinence Severity Index (FISI)
Measured: Up to 6 month after treatment initiation
The score of Hauser Ambulance Index （contains The timed 25-foot walk）
Measured: Up to 6 month after treatment initiation
The score of Hamilton Anxiety Rating Scale
Measured: Up to 6 month after treatment initiation
The score of Hamilton Depression Rating Scale
Measured: Up to 6 month after treatment initiation
The Modified Rankin Scale (mRS)
Measured: Up to 6 month after treatment initiation
The value of Quality of Life (EQ-5D-5L)
Measured: Up to 6 month after treatment initiation
The incidence of Columbia-Suicide Severity Rating Scale (C-SSRS) events
Measured: 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
Measured: 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.
Measured: Up to 6 month after treatment initiation
The value of QAlb in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Measured: Up to 6 month after treatment initiation
The value of Nfl in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Measured: Up to 6 month after treatment initiation
The value of GFAP in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to 6 month after treatment initiation
The value of sTREM2 in cerebrospinal fluid at month 1、6 compared with baseline and control group.
Measured: Up to 6 month after treatment initiation
Examination of visual function
Measured: Up to 6 month after treatment initiation
Optical coherence tomography (OCT)
Measured: Up to 6 month after treatment initiation
Translocator Protein (TSPO) PET imaging at month 1
Measured: Up to 1 month after treatment initiation
Chest computed tomography (CT) scans at month 6 and month 18
Measured: Up to 18 month after treatment initiation
Tumor marker assessment at month 6
Measured: Up to 6 month after treatment initiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2024
- Study start28 Mar 2025
- Primary completion30 Nov 2027 (estimated)
- Study completion30 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Neuromyelitis Optica Spectrum DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 69 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tongji Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- iRegene Therapeutics Co., Ltd.
- Responsible party
- Principal investigator, Wei Wang, Tongji Hospital
- Investigators
- Dai-shi Tian, MD, Principal investigator, Tongji HospoitalWei Wang, MD, Principal investigator, Tongji HospoitalChuan Qin, MD, Principal investigator, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06620809
- Sponsor's protocol ID
- NouvSoma001inNMOSD
- Status last verified
- Sep 2026
- First submitted
- 22 Sep 2024
- Last updated
- 21 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 26807122Xu 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.
PubMed 38242900