Autologous Bone Marrow Stem Cells for Children With Autism Spectrum Disorders

NCT ID: NCT01740869

Last Updated: 2025-09-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-11-30

Study Completion Date

2017-10-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Autism Autism Spectrum

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Patients

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

Group Type EXPERIMENTAL

Autologous Intrathecal Hematopoietic Stem Cells

Intervention Type 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.

Control

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.

Group Type OTHER

Observation with IDEA and CARS Scales

Intervention Type 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.

Interventions

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Autologous Intrathecal Hematopoietic Stem Cells

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.

Intervention Type BIOLOGICAL

Observation with IDEA and CARS Scales

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.

Intervention Type OTHER

Eligibility Criteria

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Inclusion 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.
Minimum Eligible Age

5 Years

Maximum Eligible Age

15 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hospital Universitario Dr. Jose E. Gonzalez

OTHER

Sponsor Role lead

Responsible Party

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M.D Laura Villarreal Martinez

Hematology Service

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Hematology Service, Hospital Universitario Dr. Jose E. Gonzalez

Monterrey, Nuevo León, Mexico

Site Status

Countries

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Mexico

References

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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.

Reference Type BACKGROUND
PMID: 22496609 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 17597540 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18555135 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 15748871 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 12202033 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18632701 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 12196638 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 16624956 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 15210522 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 15720218 (View on PubMed)

Other Identifiers

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Autism uanl

Identifier Type: OTHER

Identifier Source: secondary_id

HE12-021

Identifier Type: -

Identifier Source: org_study_id

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