NCT07243561

Prospective Clinical Study on Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes for the Treatment of Childhood Autism

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: Dongfang People's Hospital · Academic / Other
• Sex: All
• Age: 3 Years – 7 Years
• Healthy volunteers: Not accepted

Summary

This clinical study aims to evaluate whether a nasal spray containing exosomes derived from human umbilical cord mesenchymal stem cells (hUC-MSC-EXOs) can safely and effectively improve core symptoms in children aged 3-7 years with autism spectrum disorder (ASD). It is a 24-week, randomized, controlled, open-label trial. Forty pediatric patients with ASD will be randomly assigned at a 1:1 ratio to two groups: an active exosome nasal spray treatment group and a no-intervention control group. The treatment group will receive the nasal spray every other day, totaling 10 administrations throughout the study. The no-intervention control group will receive no experimental treatment but will undergo the same assessments and safety checks concurrently with the treatment group. This design aims to monitor the safety and efficacy of the hUC-MSC-EXOs nasal spray.

Conditions

• Autism Spectrum Disorder (ASD
• Prospective Study

Interventions

Type	Name	Description
BIOLOGICAL	hUC-MSC-Exos Nasal Spray	The total therapeutic dosage is set at 400 billion hUC-MSC-EXOs particles. Each nasal administration delivers 40 billion particles, with a total of 10 administrations completing the full course. The treatment is administered on an alternate-day schedule.

Timeline

• Start date: 2025-09-01
• Primary completion: 2026-07-30
• Completion: 2026-07-30
• First posted: 2025-11-24
• Last updated: 2026-04-14

Locations

1 site across 1 country: China