Nasal spray of umbilical cord mesenchymal stem-cell exosomes for children with autism
Prospective Clinical Study on Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes for the Treatment of Childhood Autism
This trial will test whether a nasal spray made from umbilical cord mesenchymal stem-cell exosomes can safely improve core autism symptoms in children aged 3 to 7.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 3 Years to 7 Years |
| Sex | All |
| Sponsor | Dongfang People's Hospital Academic / other |
| Locations | 1 site (Dongfang, Hainan) |
| Trial ID | NCT07243561 on ClinicalTrials.gov |
What this trial studies
This is a 24-week, randomized, open-label Phase 1/2 trial enrolling 40 children with ASD who meet DSM-5 or ICD-11 diagnostic criteria and have a CARS2 score of 30 or higher. Participants will be randomized 1:1 to receive an active intranasal hUC-MSC-derived exosome spray given every other day for a total of 10 administrations, or to a no-intervention control group that undergoes the same assessments and safety monitoring. The study will monitor safety outcomes and changes in core ASD symptoms over the study period. Investigators will collect clinical scales and safety labs at scheduled visits to characterize tolerability and preliminary efficacy signals.
Who should consider this trial
Good fit: Children aged 3 to 7 years with a DSM-5 or ICD-11 diagnosis of ASD, CARS2 score ≥30, who have shown no significant improvement after at least 3 months of standardized behavioral intervention and whose guardians provide informed consent are ideal candidates.
Not a fit: Children with recent epileptic seizures (within six months), autism secondary to epilepsy, cerebrovascular disease, or traumatic brain injury, those with a history of severe allergic reactions, or other excluded severe mental disorders are unlikely to benefit from this intervention.
Why it matters
Potential benefit: If successful, the treatment could reduce core social-communication and repetitive-behavior symptoms and offer a noninvasive therapeutic option for young children with ASD.
How similar studies have performed: The use of hUC-MSC-derived exosomes for ASD is largely novel with only limited preclinical work and very early-phase human data to date.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosis meets the ICD-11 ASD criteria or DSM-5 ASD clinical diagnostic standards. * No significant improvement in core symptoms was observed after ≥3 months of standardized behavioral intervention. * Score ≥30 on the CARS2, indicating mild-to-moderate or more severe autism. * Aged 3 (inclusive) to 7 (inclusive) years, regardless of gender * Voluntary participation in this clinical study, with written informed consent provided by the patient's legal guardian, and willingness to undergo examinations, treatment, and cooperate with follow-up visits. * In the investigator's judgment, the patient is capable of understanding and complying with study requirements. Exclusion Criteria: * History of severe allergic reactions. * Any severe mental disorder or other types of autism spectrum disorders. * History of epileptic seizures within the past six months. * Autism secondary to epilepsy, cerebrovascular disease, or traumatic brain injury. * Disease severity rated as normal, borderline mental disorder, or mild mental disorder on the Clinical Global Impression scale. * Moderate or severe extrapyramidal symptoms or tardive dyskinesia. * Severe self-injurious behavior. * Active systemic or severe localized infections, including human immunodeficiency virus, syphilis, and hepatitis. * Autoimmune diseases. * Major organ impairment. * Severe pulmonary or hematological diseases, malignancies, or immunodeficiency. * Concurrent treatments that may interfere with the safety and efficacy evaluation of stem cell therapy. * Participation in other clinical trials within the past three months. * Other clinical conditions deemed by investigators as unsuitable for study inclusion.
Where this trial is running
Dongfang, Hainan
- Dongfang People's Hospital — Dongfang, Hainan, China (Recruiting)
Study contacts
- Study coordinator: Zhongmin Liu
- Email: dfsrmyykjk@126.com
- Phone: +86-0898-38932608
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.