hUC-MSC-Exo Therapy for Autoimmune Encephalitis

NCT07131683 | Not Yet Recruiting Phase 1 DMC

• 2 other identifiers: KS2025069BRWEP2024W022010110
• Study Type: interventional
• Target: 38
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a phase I/IIa study to investigate the safety and preliminary efficacy of intranasal admnistration of human umbilical mesenchymal stem cell-derived exosome (hUC-MSC-Exo) for patients with autoimmune encephalitis.

Conditions

Autoimmune Encephalitis

Keywords

mesenchymal stem cell; exosome; autoimmune encephalitis; safety; efficacy

Outcome Measures

Primary Outcomes (2)

• DLT events

  Drug-related dose limiting toxicity (DLT)

  Within 24 weeks after drug administration

• mRS

  modified Ranking Scale (mRS), which ranges from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.

  24 weeks ±10 days

Secondary Outcomes (9)

• AE and SAE

  Within 24 weeks after drug administration

• mRS

  5 weeks (±3 days), 12 weeks (±5 days)

• Percentage of mRS 0-2

  5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)

• Percentage of mRS improvement ≧ 1

  5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)

• CASE score change compared to baseline

  5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)

Study Arms (2)

Exosome group

EXPERIMENTAL

Biological: Human umbilical cord mesenchymal stem cell derived exosomes

Control group

PLACEBO COMPARATOR

Other: Placebo Control

Interventions

Human umbilical cord mesenchymal stem cell derived exosomes BIOLOGICAL

Nasal spray of hUC-MSC-Exo (low dose: 2.5×10\^10 particles, mid-dose: 5.0×10\^10 particles, high-dose: 1.0×10\^11 particles), once per day for 7 days, then once per week for 3 weeks

Also known as: hUC-MSC-Exo

Exosome group

Placebo Control OTHER

Nasal spray of placebo control, once per day for 7 days, then once per week for 3 weeks

Also known as: hUC-MSC-Exo mimics

Control group

Eligibility Criteria

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

You may qualify if:

• Aged 18-65 years, both male and female are eligible;
• Diagnosis of autoimmune encephalitis within 3 months of onset (meeting the 2016 Graus and Dalmau diagnostic criteria), with positive serum/cerebrospinal fluid anti-NMDAR antibodies or anti-LGI1 antibodies;
• Modified Rankin Scale (mRS) score ≥ 2 at enrollment;
• The subject or legally authorized representative is able to sign the informed consent form;
• Subjects of childbearing potential must agree to practice strict contraception during the study period.

You may not qualify if:

• Pre-morbid modified Rankin Scale (mRS) score ≥ 2;
• Known allergy to any component of the investigational product or history of severe allergic reactions;
• Presence of neurological or psychiatric disorders (e.g., cerebrovascular disease, Parkinson's disease, severe depression) deemed by the investigator to potentially impair trial participation or study assessments;
• Severe cardiovascular diseases (e.g., congestive heart failure, severe arrhythmia, myocardial infarction)
• Hepatic diseases (e.g., cirrhosis)
• Renal diseases (e.g., requiring hemodialysis or peritoneal dialysis)
• Hematological diseases (e.g., hemophilia with bleeding tendency)
• Endocrine disorders (e.g., poorly controlled diabetes with blood glucose \>16.8 mmol/L or \<2.8 mmol/L, or with severe complications)
• Immune system disorders (active or uncontrolled systemic autoimmune diseases, primary/secondary immunodeficiency)
• Malignancies;
• Anatomical nasal abnormalities, nasal mucosal damage, severe rhinitis, or other nasal conditions affecting drug administration;
• Requiring nasogastric tube placement;
• Organ function meeting any of the following criteria:
• Absolute neutrophil count (ANC) \<1.5×10⁹/L, platelets (PLT) \<100×10⁹/L, hemoglobin (Hb) \<90 g/L
• Aspartate aminotransferase (AST) \>2.5×ULN and/or alanine aminotransferase (ALT) \>2.5×ULN, total bilirubin (TBIL) \>1.5×ULN

Sponsors & Collaborators

• Xuanwu Hospital, Beijing: lead
• ECHO Biotech: collaborator

MeSH Terms

Conditions

Autoimmune Diseases of the Nervous System

Condition Hierarchy (Ancestors)

Nervous System Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle investigator

Study Record Dates

• First Submitted: August 7, 2025
• First Posted: August 20, 2025
• Study Start: November 1, 2025
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): August 31, 2027
• Last Updated: August 20, 2025

Record last verified: 2025-08