Evaluating NouvSoma001 for treating 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 study is testing a new treatment called NouvSoma001 to see if it can help people with Neuromyelitis Optica Spectrum Disorders feel better and stay safe while using it.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 69 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Tongji Hospital (Academic / other)
• Locations: 1 site (Wuhan, Hubei)
• Trial ID: NCT06620809 on ClinicalTrials.gov

What this trial studies

This clinical trial is a single-center, randomized, open-label, placebo-controlled, dose-escalation study aimed at assessing the safety, tolerability, and efficacy of NouvSoma001, which consists of extracellular vesicles derived from human-induced neural stem cells. The trial is divided into two parts: the first part involves a dose-escalation design with three cohorts receiving increasing doses of the treatment, while the second part will randomize participants to either the treatment or placebo group based on the findings from the first part. The study will enroll a total of 69 participants, with careful monitoring for dose-limiting toxicities during the initial phase.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 to 65 who have recently experienced symptoms of Neuromyelitis Optica Spectrum Disorders and test positive for AQP4 antibodies.

Not a fit: Patients with significant laboratory abnormalities or those who do not meet the diagnostic criteria for Neuromyelitis Optica Spectrum Disorders may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients suffering from Neuromyelitis Optica Spectrum Disorders.

How similar studies have performed: While the use of extracellular vesicles in this context is relatively novel, similar approaches in regenerative medicine have shown promise in other conditions.

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.

Where this trial is running

Wuhan, Hubei

• Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology — Wuhan, Hubei, China (Recruiting)

Study contacts

• Principal investigator: Dai-shi Tian, MD — Tongji Hospoital
• Study coordinator: Chuan Qin, MD

Conditions Neuromyelitis Optica Spectrum Disorders; extracelluar vesicles