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
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.
Design
Phase II clinical trial, safety evaluation, single group, no control
Settings and conduct
Five autistic children, aged 3 to 5 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.
Participants/Inclusion and exclusion criteria
Children ages two to 5 years 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.
Intervention groups
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.
Main outcome variables
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
Reason for update
Acronym
MSCA
IRCT registration information
IRCT registration number:IRCT20200217046526N3
Registration date:2020-04-15, 1399/01/27
Registration timing:prospective
Last update:2020-04-15, 1399/01/27
Update count:0
Registration date
2020-04-15, 1399/01/27
Registrant information
Name
Masoumeh Nouri
Name of organization / entity
Royan Stem Cell Technology Co
Country
Iran (Islamic Republic of)
Phone
+98 21 2763 5512
Email address
m.nouri@rsct.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2020-06-21, 1399/04/01
Expected recruitment end date
2020-07-22, 1399/05/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
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
Public title
Stem Cell Therapy for Autism
Purpose
Treatment
Inclusion/Exclusion criteria
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
Age
From 36 months old to 60 months old
Gender
Both
Phase
2
Groups that have been masked
No information
Sample size
Target sample size:
5
Randomization (investigator's opinion)
N/A
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Single
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
ACECR Biomedical Research Ethics Committee
Street address
No. 1270, Enghelab St., Tehran., District 11., Tehran Province., Iran
City
Tehran
Province
Tehran
Postal code
14155-4364
Approval date
2020-02-23, 1398/12/04
Ethics committee reference number
IR.ACECR.REC.1398.003
Health conditions studied
1
Description of health condition studied
Autism Spectrum Disorder
ICD-10 code
ICD-10 code description
F84.0
Primary outcomes
1
Description
ََAdverse events assesment
Timepoint
َAt the time of each injection, 24 hours after each injection, 7-10 days after each injection, 3 month after the final injection
Method of measurement
Common Terminology Criteria for Adverse Events (CTCAE) Version 4
Secondary outcomes
1
Description
Change in Childhood Autism Rating Scale, CARS
Timepoint
Before injection, 7-10 days after each injection, 3 months after the final injection
Method of measurement
Childhood Autism Rating Scale form
2
Description
Change in Autism Treatment Evaluation Checklist
Timepoint
Before injection, 7-10 days after each injection, 3 months after the final injection
Method of measurement
Autism Treatment Evaluation Checklist
3
Description
Change in the Behavior Problems Inventory
Timepoint
Before injection, 7-10 days after each injection, 3 months after the final injection
Method of measurement
The Behavior Problems Inventory Checklist
4
Description
Change in Electroencephalogram
Timepoint
Before injection, 7-10 days after each injection, 3 months after the final injection
Method of measurement
Change in EEG waves
5
Description
Assess the quality of life base on Short-form 36
Timepoint
Before injection, 7-10 days after each injection, 3 months after the final injection
Method of measurement
SF 36 questionnaire
Intervention groups
1
Description
Intervention group: 5 autistic children receiving mesenchymal cells intrathecally at one month intervals.
Category
Treatment - Other
Recruitment centers
1
Recruitment center
Name of recruitment center
Roozbeh Hospital
Full name of responsible person
Hadi Zarafshan
Street address
No. 606, South Karagar St., District 11
City
Tehran
Province
Tehran
Postal code
13337159140
Phone
+98 21 5541 9151
Email
zarafshan84@gmail.com
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Mohammad Ali Sahraian
Street address
ImamKhomeini Ave., Hasan Abad Sq., Sina Hospital
City
تهران
Province
Tehran
Postal code
١١٣٦٧٤٦٩١١
Phone
+98 21 6670 6142
Email
msahrai@tums.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?