Safety and Efficacy of Stem Cell Therapy in Patients With Autism

Study Overview
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Brief Summary
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Autism is one of those disorders in Autism spectrum disorders (ASD), which characterized by social interaction abnormalities, impaired verbal and non-verbal communication, and repetitive, obsessive behavior, while the therapeutic effect of current treatments remains limited progress .Neural hypoperfusion and immune deregulation are the two key pathologies associated with Autism. Human umbilical cord mesenchymal stem cells (hUC-MSCs) and human cord blood mononuclear cells (hCB-MNCs) have been shown to have the ability to modulate the immune response and enhance angiogenesis, suggesting the novel and promising therapeutic strategy. In this study, the safety and efficacy of hUC-MSCs and hCB-MNCs transplantation will be evaluated in patients with Autism.

Detailed Description
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To investigate the safety and efficacy of human cord blood mononuclear cells and human umbilical cord mesenchymal stem cells transplantation in patients of Autism.

Official Title
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Phase Ι/Π Study of Stem Cell Therapy in Patients With Autism

Conditions
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Autism

Intervention / Treatment
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* Biological: human cord blood mononuclear cells
* Biological: human cord blood mononuclear cells and human umbilical cord mesenchymal stem cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children between the ages of 3 and 12 years. DSM-IV diagnosis of Autistic Disorder. Total score of CARS ≥ 30. Parents or legal guardian willing to sign the ICF. Exclusion Criteria: Any history of hypersensitivity to serum products, or other known drug and food allergy. History of prior or current DSM-IV psychotic disorder (e.g., schizophrenia, bipolar disorder, other psychosis), Pervasive Developmental Disorder not otherwise specified (PDD NOS), Asperger's, or Rett's. History of Epileptic seizure activity in the past 6 months. Autism caused by seizure disorders (active), cerebrovascular disease or brain trauma. The global autism ratings are assessed as being absent, minimal or mild. Existing moderate or severe extrapyramidal symptoms (EPS) or history of tardive dyskinesia. Subjects who have displayed significant self-injurious behavior (children who have caused visible harm to themselves). HIV+ Acute and chronic hepatitis. Autoimmune disease, e.g. lupus erythematosus, multiple sclerosis. Severe pulmonary and hematological disease, malignancy or hypo-immunity. Currently undertaking other treatment that may affect the safety/efficacy of stem cells. Enrollment in other trials in the last 3 months. Other criteria the investigator consider improper for inclusion.

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Rehabilitation plus hCB-MNCs treatment<br>Participants will be given rehabilitation therapy plus human cord blood mononuclear cells transplantation with a 6 months follow-up. | Biological: human cord blood mononuclear cells<br>* Participants will be given rehabilitation therapy plus hCB-MNCs transplantation.<br>* Other names: Group 1;|
| Experimental: Rehabilitation plus hCB-MNCs and hUC-MSCs therapy<br>Participants will be given rehabilitation therapy plus combination of hCB-MNCs together with hUC-MSCs transplantation with a 6 months follow-up. | Biological: human cord blood mononuclear cells and human umbilical cord mesenchymal stem cells<br>* Participants will be given rehabilitation therapy plus and hCB-MNCs and hUC-MSCs transplantation.<br>* Other names: Group 2;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Childhood Autism Rating Scale，CARS |  | 6 months after treatment | 
| Clinical Global Impression Scale，CGI |  | 6 months after treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Aberrant Behavior Checklist，ABC |  | 6 months after treatment | 
| Adverse Event and Serious Adverse Event |  | 6 months after treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Autism, human cord blood mononuclear cells, human umbilical cord mesenchymal stem cells