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AutismPhase 3AutismNCT01740869

Autologous Bone Marrow Stem Cells for Children With Autism Spectrum Disorders

A Phase 3 study testing Autologous Intrathecal Hematopoietic Stem Cells and Observation with IDEA and CARS Scales in people with Autism and Autism Spectrum, run by Hospital Universitario Dr. Jose E. Gonzalez at 1 site in Mexico.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 5 to 15 years
Conditions the study is for
Autism, Autism Spectrum
What is being tested?
Autologous Intrathecal Hematopoietic Stem Cells and Observation with IDEA and CARS Scales
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
1 siteMexico
Study dates
Nov 2012to Oct 2017
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 15 years
  • All sexes
  • No healthy volunteers
  • Child

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 locations and contacts

1 site in 1 country.

Mexico1 site

  • Hematology Service, Hospital Universitario Dr. Jose E. Gonzalez

    Monterrey, Nuevo León, 64460

Other trials in Mexico

About this study

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.

Read the detailed 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.

Being tested
Autologous Intrathecal Hematopoietic Stem Cells, Observation with IDEA and CARS Scales
Keywords
autism, autism spectrum, stem cells, children
Medical subject headings
Autistic Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Autologous Intrathecal Hematopoietic Stem CellsBiological

    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.

  • Observation with IDEA and CARS ScalesOther

    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.

Study groups in NCT01740869
Study groupRoleWhat they receive
PatientsExperimentalChildren with autism spectrum disorder who will receive intrathecal infusion of autologous CD34+ hematopoietic stem cells after G-CSF stimulation and bone marrow harvest.
Receives: Biological: Autologous Intrathecal Hematopoietic Stem Cells
ControlOtherChildren 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.
Receives: Other: Observation with IDEA and CARS Scales

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Baseline, 30 days, and 180 days

Secondary outcomes (1)

  1. Change in IDEA scale score

    IDEA scale will assess developmental and social abilities in participants. Measurements will be taken at baseline, 30 days, and 180 days to evaluate the effect of treatment on developmental progress.

    Measured: Baseline, 30 days, and 180 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Dec 2012 (estimated)
  2. Study startNov 2012
  3. Primary completionNov 2016
  4. Study completionOct 2017

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Crossover
Masking
None (open label)
Enrolment
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01740869
Sponsor's protocol ID
HE12-021
Other IDs
Autism uanl
Acronym
Autism
Status last verified
Mar 2025
First submitted
22 Nov 2012
Last updated
16 Sep 2025
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • 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.

    PubMed 22496609

  • 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.

    PubMed 17597540

  • 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.

    PubMed 18555135

  • 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.

    PubMed 15748871

  • 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.

    PubMed 12202033

  • 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.

    PubMed 18632701

  • 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.

    PubMed 12196638

  • 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.

    PubMed 16624956

  • 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.

    PubMed 15210522

  • 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.

    PubMed 15720218

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Autologous Intrathecal Hematopoietic Stem Cells and Observation with IDEA and CARS Scales in people with Autism and Autism Spectrum, run by Hospital Universitario Dr. Jose E. Gonzalez at 1 site in Mexico.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Mexico. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in Childhood Autism Rating Scale (CARS) score (time frame: Baseline, 30 days, and 180 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01740869, which also lists 10 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2025.

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