Clinical trial NCT01343511: Safety and Efficacy of Stem Cell Therapy in Patients With Autism
Official title: Phase Ι/Π Study of Stem Cell Therapy in Patients With Autism
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Autism
Keywords: Autism
human cord blood mononuclear cells
human umbilical cord mesenchymal stem cells
Lead sponsor: Shenzhen Beike Bio-Technology Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Shandong Jiaotong Hospital
name: Association for the Handicapped Of Jinan
First posted: 2011-04-28
Enrollment: 37
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Eligibility criteria: 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.

Primary outcomes:
measure: Childhood Autism Rating Scale，CARS
timeFrame: 6 months after treatment
measure: Clinical Global Impression Scale，CGI
timeFrame: 6 months after treatment
Secondary outcomes:
measure: Aberrant Behavior Checklist，ABC
timeFrame: 6 months after treatment
measure: Adverse Event and Serious Adverse Event
timeFrame: 6 months after treatment
Other outcomes:
Locations:
city: Jinan
country: China
facility: Shandong Jiaotong Hospital
state: Shandong
Study officials:
affiliation: Shandong Jiaotong Hospital
name: Yongtao Lv
role: PRINCIPAL_INVESTIGATOR
References:
citation: Liu M, Sun LW, LV YT, Huan Y, Ge RC, Cao YL, Guo CQ, Chen XW. Stem cells for treatment of autism: Safety and efficacy. Zhongguo Zuzhi Gongcheng Yanjiu yu Linchuang Kangfu. 2010;14(32): 5967-5970.
citation: Lv YT, Zhang Y, Liu M, Qiuwaxi JN, Ashwood P, Cho SC, Huan Y, Ge RC, Chen XW, Wang ZJ, Kim BJ, Hu X. Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism. J Transl Med. 2013 Aug 27;11:196. doi: 10.1186/1479-5876-11-196.