Autologous Bone Marrow Stem Cells for Children With Autism Spectrum Disorders

NCT01740869 | Completed Phase 3 DMC

• 2 other identifiers: HE12-021Autism uanl
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Autism; Autism Spectrum

Keywords

autism; autism spectrum; stem cells; children

Outcome Measures

Primary Outcomes (1)

• Change in Childhood Autism Rating Scale (CARS) score

  CARS scale will be used to evaluate changes in autistic behavior and social interaction after treatment. Participants will be assessed at baseline, 30 days, and 180 days to measure improvement following the intrathecal infusion of autologous CD34+ hematopoietic stem cells.

  Baseline, 30 days, and 180 days

Secondary Outcomes (1)

• Change in IDEA scale score

  Baseline, 30 days, and 180 days

Study Arms (2)

Patients

EXPERIMENTAL

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

Biological: Autologous Intrathecal Hematopoietic Stem Cells

Control

OTHER

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.

Other: Observation with IDEA and CARS Scales

Interventions

Autologous Intrathecal Hematopoietic Stem Cells BIOLOGICAL

Patients will be stimulated with granulocyte colony-stimulating factor (G-CSF) for 3 consecutive days. Bone marrow will be harvested, processed to isolate CD34+ hematopoietic stem cells and reduce red blood cells. An inoculum of 5-10 mL will then be infused intrathecally into the cerebrospinal fluid.

Patients

Observation with IDEA and CARS Scales OTHER

Participants will be observed for 6 months without active intervention. They will be evaluated using the Childhood Autism Rating Scale (CARS) and the IDEA scale at baseline, 30 days, and 180 days. After 6 months, participants may crossover to the experimental arm.

Control

Eligibility Criteria

• Age: 5 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

Sponsors & Collaborators

• Hospital Universitario Dr. Jose E. Gonzalez: lead

Study Sites (1)

Hematology Service, Hospital Universitario Dr. Jose E. Gonzalez

Monterrey, Nuevo León, 64460, Mexico

Location

Related Publications (10)

• Siniscalco D, Sapone A, Cirillo A, Giordano C, Maione S, Antonucci N. Autism spectrum disorders: is mesenchymal stem cell personalized therapy the future? J Biomed Biotechnol. 2012;2012:480289. doi: 10.1155/2012/480289. Epub 2012 Feb 13.

  PMID: 22496609 BACKGROUND

• Ichim TE, Solano F, Glenn E, Morales F, Smith L, Zabrecky G, Riordan NH. Stem cell therapy for autism. J Transl Med. 2007 Jun 27;5:30. doi: 10.1186/1479-5876-5-30.

  PMID: 17597540 BACKGROUND

• Mehta T, Feroz A, Thakkar U, Vanikar A, Shah V, Trivedi H. Subarachnoid placement of stem cells in neurological disorders. Transplant Proc. 2008 May;40(4):1145-7. doi: 10.1016/j.transproceed.2008.03.026.

  PMID: 18555135 BACKGROUND

• Hayashi T, Iwai M, Ikeda T, Jin G, Deguchi K, Nagotani S, Zhang H, Sehara Y, Nagano I, Shoji M, Ikenoue T, Abe K. Neural precursor cells division and migration in neonatal rat brain after ischemic/hypoxic injury. Brain Res. 2005 Mar 15;1038(1):41-9. doi: 10.1016/j.brainres.2004.12.048.

  PMID: 15748871 BACKGROUND

• Nakatomi H, Kuriu T, Okabe S, Yamamoto S, Hatano O, Kawahara N, Tamura A, Kirino T, Nakafuku M. Regeneration of hippocampal pyramidal neurons after ischemic brain injury by recruitment of endogenous neural progenitors. Cell. 2002 Aug 23;110(4):429-41. doi: 10.1016/s0092-8674(02)00862-0.

  PMID: 12202033 BACKGROUND

• Goldman SA, Schanz S, Windrem MS. Stem cell-based strategies for treating pediatric disorders of myelin. Hum Mol Genet. 2008 Apr 15;17(R1):R76-83. doi: 10.1093/hmg/ddn052.

  PMID: 18632701 BACKGROUND

• Rempe DA, Kent TA. Using bone marrow stromal cells for treatment of stroke. Neurology. 2002 Aug 27;59(4):486-7. doi: 10.1212/wnl.59.4.486. No abstract available.

  PMID: 12196638 BACKGROUND

• Felling RJ, Snyder MJ, Romanko MJ, Rothstein RP, Ziegler AN, Yang Z, Givogri MI, Bongarzone ER, Levison SW. Neural stem/progenitor cells participate in the regenerative response to perinatal hypoxia/ischemia. J Neurosci. 2006 Apr 19;26(16):4359-69. doi: 10.1523/JNEUROSCI.1898-05.2006.

  PMID: 16624956 BACKGROUND

• Gordon PH, Yu Q, Qualls C, Winfield H, Dillon S, Greene PE, Fahn S, Breeze RE, Freed CR, Pullman SL. Reaction time and movement time after embryonic cell implantation in Parkinson disease. Arch Neurol. 2004 Jun;61(6):858-61. doi: 10.1001/archneur.61.6.858.

  PMID: 15210522 BACKGROUND

• Kulbatski I, Mothe AJ, Nomura H, Tator CH. Endogenous and exogenous CNS derived stem/progenitor cell approaches for neurotrauma. Curr Drug Targets. 2005 Feb;6(1):111-26. doi: 10.2174/1389450053345037.

  PMID: 15720218 BACKGROUND

MeSH Terms

Conditions

Autistic Disorder

Interventions

Observation

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Methods; Investigative Techniques

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Hematology Service

Study Record Dates

• First Submitted: November 22, 2012
• First Posted: December 4, 2012
• Study Start: November 1, 2012
• Primary Completion: November 1, 2016
• Study Completion: October 1, 2017
• Last Updated: September 16, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

ME (1)