The Safety and Efficacy of NouvSoma001 in Neuromyelitis Optica Spectrum Disorders

NCT ID: NCT06620809

Last Updated: 2026-01-21

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 69 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-03-28
• Study Completion Date: 2027-11-30

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.

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.

Conditions

Neuromyelitis Optica Spectrum Disorders

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

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

Intervention Type: DRUG

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

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.

Group Type: PLACEBO_COMPARATOR

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

Intervention Type: DRUG

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

Interventions

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）

Intervention Type: DRUG

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

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

Intervention Type: DRUG

Other Intervention Names

NouvSoma001; NouvSoma001

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

iRegene Therapeutics Co., Ltd.

INDUSTRY

Sponsor Role: collaborator

Tongji Hospital

OTHER

Sponsor Role: lead

Responsible Party

Wei Wang

Prof.

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Dai-shi Tian, MD

Role: PRINCIPAL_INVESTIGATOR

Tongji Hospoital

Wei Wang, MD

Role: PRINCIPAL_INVESTIGATOR

Tongji Hospoital

Chuan Qin, MD

Role: PRINCIPAL_INVESTIGATOR

Tongji Hospital

Locations

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

Wuhan, Hubei, China

Site Status: RECRUITING

Countries

China

Central Contacts

Chuan Qin, MD

Role: CONTACT

qinchuan712@126.com

86-27-83663337

Chuan Qin, MD

Role: CONTACT

chuanqin@tjh.tjmu.edu.cn

86-27-83663332

Facility Contacts

Chuan Qin, MD

Role: primary

86-27-83663337

Chuan Qin, MD

Role: backup

86-27-83663332

References

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.

Reference Type: BACKGROUND

PMID: 26807122 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 38242900 (View on PubMed)

Other Identifiers

NouvSoma001inNMOSD

Identifier Type: -

Identifier Source: org_study_id