Protocol summary
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Study aim
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This study aims to evaluate the safety of three intrathecal injections (at one-month intervals) of human umbilical cord tissue-derived mesenchymal stromal cells, at a fixed dose of 20 million allografts, to reduce symptoms and Improve function Children with Autism.
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Design
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Phase II clinical trial, safety evaluation, single group, no control
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Settings and conduct
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Five autistic children, aged 3 to 7 years, referred to Roozbeh Hospital who met the inclusion criteria were included in the intervention group. The subjects will receive three intrathecal injections (at one-month intervals) of, human umbilical cord tissue-derived mesenchymal stromal cells, at a fixed dose of 20 million.
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Participants/Inclusion and exclusion criteria
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Children aged between 3 to 7 years diagnosed with Autism (level II) will be eligible to participate.
Confirmed clinical DSM-5 diagnosis of Autism using the DSM-5 Checklist with level II;
History of unstable or uncontrolled seizure disorder, History of prior cell therapy or participation in other clinical trials.
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Intervention groups
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One group; subjects will receive three intrathecal injections (at one-month intervals) of, human umbilical cord tissue-derived mesenchymal stromal cells, at a fixed dose of 20 million.
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Main outcome variables
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Determination of side effects; Symptom severity based on CARS; Impact on evolution based on the ATEC form; Change in severity of behavioral disorder based on BPI-S form; EEG-based changes in electroencephalographic waves; Impact on Mental Health and Parental Stress in Children with Autism Based on SF36 Form.
General information
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Reason for update
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Due to challenges in patient recruitment, the eligible age range for children diagnosed with Autism Spectrum Disorder has been revised from 3–5 years to 3–7 years. This amendment was approved by the Central Ethics Committee of the Academic Center for Education, Culture, and Research (ACECR).
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Acronym
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MSCA
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IRCT registration information
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IRCT registration number:
IRCT20200217046526N3
Registration date:
2020-04-15, 1399/01/27
Registration timing:
prospective
Last update:
2025-08-15, 1404/05/24
Update count:
1
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Registration date
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2020-04-15, 1399/01/27
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Registrant information
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Recruitment status
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Recruitment complete
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Funding source
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Expected recruitment start date
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2020-06-21, 1399/04/01
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Expected recruitment end date
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2020-07-22, 1399/05/01
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Actual recruitment start date
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empty
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Actual recruitment end date
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empty
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Trial completion date
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empty
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Scientific title
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A phase I trial evaluating the safety of 3 Times Intratechal Injection of the-MSC, an Allogenic Umbilical cord-derived Mesenchymal Stromal Cell Product, in Children with Autism Spectrum Disorder
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Public title
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Stem Cell Therapy for Autism
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Purpose
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Treatment
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Inclusion/Exclusion criteria
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Inclusion criteria:
Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist with level 2
Parental consent
Stable on current psychiatric medication regimen (dose and dosing schedule) for at least 2 months prior to infusion of study product
Exclusion criteria:
Review of medical records indicates Autism diagnosis not likely
Presence of any concomitant systemic disease (active infection, any malignancy,...), Central Nervous System Infection, people with Nervous System Inflammation, Cerebral palsy
Presence of any psychiatric disorders such as Asperger's syndrome, Depression, Bipolar disorder, Schizophrenia, Tourette's syndrome
History of major surgery in the last 30 days
History of allogeneic tissue transplantation
History of active seizure disorder over the past 6 months
History of a primary immunodeficiency disorder, HIV
Records indicate that the child has a known genetic syndrome related to Autism
History of allergic disease
Participate in other clinical trial studies
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Age
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From 36 months old to 84 months old
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Gender
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Both
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Phase
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1
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Groups that have been masked
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No information
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Sample size
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Target sample size:
5
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Randomization (investigator's opinion)
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N/A
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Randomization description
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Blinding (investigator's opinion)
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Not blinded
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Blinding description
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Placebo
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Not used
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Assignment
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Single
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Other design features
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Ethics committees
1
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Ethics committee
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Approval date
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2020-02-23, 1398/12/04
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Ethics committee reference number
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IR.ACECR.REC.1398.003
Health conditions studied
1
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Description of health condition studied
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Autism Spectrum Disorder
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ICD-10 code
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ICD-10 code description
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F84.0
Primary outcomes
1
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Description
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ََAdverse events assesment
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Timepoint
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َAt the time of each injection, 24 hours after each injection, 7-10 days after each injection, 3 month after the final injection
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Method of measurement
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Common Terminology Criteria for Adverse Events (CTCAE) Version 4
Secondary outcomes
1
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Description
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Change in Childhood Autism Rating Scale, CARS
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Timepoint
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Before injection, 7-10 days after each injection, 3 months after the final injection
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Method of measurement
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Childhood Autism Rating Scale form
2
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Description
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Change in Autism Treatment Evaluation Checklist
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Timepoint
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Before injection, 7-10 days after each injection, 3 months after the final injection
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Method of measurement
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Autism Treatment Evaluation Checklist
3
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Description
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Change in the Behavior Problems Inventory
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Timepoint
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Before injection, 7-10 days after each injection, 3 months after the final injection
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Method of measurement
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The Behavior Problems Inventory Checklist
4
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Description
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Change in Electroencephalogram
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Timepoint
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Before injection, 7-10 days after each injection, 3 months after the final injection
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Method of measurement
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Change in EEG waves
5
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Description
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Assess the quality of life base on Short-form 36
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Timepoint
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Before injection, 7-10 days after each injection, 3 months after the final injection
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Method of measurement
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SF 36 questionnaire
Intervention groups
1
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Description
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Intervention group: 5 autistic children receiving mesenchymal cells intrathecally at one month intervals.
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Category
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Treatment - Other
1
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Sponsor
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Grant name
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Grant code / Reference number
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Is the source of funding the same sponsor organization/entity?
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Yes
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Title of funding source
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Tehran University of Medical Sciences
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Proportion provided by this source
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1
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Public or private sector
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Public
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Domestic or foreign origin
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Domestic
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Category of foreign source of funding
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empty
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Country of origin
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Type of organization providing the funding
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Academic
2
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Sponsor
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Grant name
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Grant code / Reference number
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Is the source of funding the same sponsor organization/entity?
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Yes
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Title of funding source
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Royan Stem Cell Technology Co.
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Proportion provided by this source
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100
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Public or private sector
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Private
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Domestic or foreign origin
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Domestic
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Category of foreign source of funding
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empty
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Country of origin
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Type of organization providing the funding
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Industry
Sharing plan
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Deidentified Individual Participant Data Set (IPD)
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Yes - There is a plan to make this available
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Study Protocol
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Undecided - It is not yet known if there will be a plan to make this available
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Statistical Analysis Plan
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Undecided - It is not yet known if there will be a plan to make this available
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Informed Consent Form
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Undecided - It is not yet known if there will be a plan to make this available
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Clinical Study Report
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Undecided - It is not yet known if there will be a plan to make this available
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Analytic Code
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Undecided - It is not yet known if there will be a plan to make this available
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Data Dictionary
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Undecided - It is not yet known if there will be a plan to make this available
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Title and more details about the data/document
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All collected deidentified IPD can be shared
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When the data will become available and for how long
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6 months after publication
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To whom data/document is available
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Researchers and clinicians
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Under which criteria data/document could be used
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Planning of similar studies in other academic centers
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From where data/document is obtainable
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eMail
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What processes are involved for a request to access data/document
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1-2 months after request during
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Comments
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