NCT07131683

hUC-MSC-Exo Therapy for Autoimmune Encephalitis

Human Umbilical Cord Mesenchymal Stem Cell-derived ExoSomes for Autoimmune Encephalitis: a Phase I/IIa Clinical Trial (MESAE)

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 38 (estimated)
• Sponsor: Xuanwu Hospital, Beijing · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Dose Escalation Phase: A multicenter, single-arm, open-label study will be conducted to evaluate the safety, tolerance, and dose exploration of multiple administrations of hUC-MSC-Exo for treating AE. Three dose cohorts (2.5×10¹⁰, 5.0×10¹⁰, and 1.0×10¹¹ particles) will be enrolled with 3-6 subjects each. Administration will be intranasal, once daily for 7 consecutive days, followed by once weekly for 3 consecutive weeks, resulting in a total treatment period of 4 weeks. After the last subject in each cohort completes the final dose and undergoes a 21-day safety assessment, a decision will be made regarding progression to the next higher dose cohort for further evaluation of safety and tolerance. The maximal tolerance dose (MTD) will be determined. Case Expansion Phase: A multicenter, randomized, double-blind, placebo-controlled study will enroll 20 subjects randomly assigned to either the experimental group (exosome group) or the control group (exosome mimetic group) in a 1:1 ratio. The dosage for the experimental group will be determined by the Data Safety Monitoring Board (DSMB) based on the safety and efficacy data obtained during the dose exploration phase.

Conditions

• Autoimmune Encephalitis

Interventions

Type	Name	Description
BIOLOGICAL	Human umbilical cord mesenchymal stem cell derived exosomes	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
OTHER	Placebo Control	Nasal spray of placebo control, once per day for 7 days, then once per week for 3 weeks

Timeline

• Start date: 2025-11-01
• Primary completion: 2027-06-30
• Completion: 2027-08-31
• First posted: 2025-08-20
• Last updated: 2025-08-20