Autologous Bone Marrow Stem Cells for Children With Autism Spectrum Disorders

Hospital Universitario Dr. Jose E. Gonzalez

Status and phase: Completed; Phase 3

Conditions: Autism Spectrum; Autism

Treatments: Other: Observation with IDEA and CARS Scales; Biological: Autologous Intrathecal Hematopoietic Stem Cells

Study type: Interventional

Funder types: Other

Identifiers

NCT01740869

Autism uanl (Other Identifier)

HE12-021

Details and patient eligibility

About

The purpose of this study is to determine whether the plasticity of autologous intrathecal hematopoietic cells would improve the neurologic and the social skills of pediatric patients with autism spectrum disorders.

Full description

There is accumulating evidence that shows that the administration of hematopoietic cells into the brain in the patients with spectrum autism could help in the physiopathology of the illness.

It has been found that after introducing hematopoietic cells in the subarachnoid space of the spinal cord, these cells may be transported through the cerebrospinal fluid and can be delivered more efficiently to the injured area, when compared to the intravenous route.

Patients will be stimulated for 3 consecutive days with granulocyte colony stimulating factor (G-CSF) and then their bone marrow will be harvested according to their weight. Bone marrow will be processed in order to obtain CD34+ cells and minimize the amount of red blood cells. An inoculum of 5 to 10mL of stem cells will be infused intrathecally. Patients will be evaluated with two scales "CARS" and the "IDEA" also we will check the clinical history. On days 0, 30 and 180.

Enrollment: 30 patients

Sex: All

Ages: 5 to 15 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria: Children aged 5 to 15 years, Diagnosis of Autism Spectrum Disorder (ASD) confirmed by DSM-5 criteria, Ability to comply with study procedures, including assessments with CARS and IDEA scales, Written informed consent obtained from parents or legal guardians.

Exclusion Criteria: Presence of severe neurological disorders other than ASD, History of immunodeficiency or hematologic disorders, Active infection at the time of enrollment, Previous treatment with intrathecal stem cells, Any medical condition that, in the investigator's judgment, would make participation unsafe.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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30 participants in 2 patient groups

Patients

Experimental group

Description:

Children with autism spectrum disorder who will receive intrathecal infusion of autologous CD34+ hematopoietic stem cells after G-CSF stimulation and bone marrow harvest.

Treatment:

Biological: Autologous Intrathecal Hematopoietic Stem Cells

Control

Other group

Description:

Children with autism spectrum disorder who will be observed for 6 months and evaluated with CARS and IDEA scales, with option to crossover to the experimental arm afterwards.

Treatment:

Other: Observation with IDEA and CARS Scales