To evaluate the efficacy of stem cell therapy in improving the Scale for Assessment and Rating of Ataxia(SARA) score in patients with Ataxia-Telangiectasia at 12 month follow up
Design
this study is a single center non randomized, phase1/2 pilot clinical trial to evaluate the safety and efficacy of allogenic mesenchymal stem cell transplantation in 5 patients with Ataxia telangiectasia
Settings and conduct
Study location: Pastor Hospital
Method: IV and Intrathecal stem cell infusion in day care unit 24 hours monitoring and periodic clinic assessment
Participants/Inclusion and exclusion criteria
Inclusion criteria;
Patient with a confirmed diagnosis of Ataxia-Telangiectasia based on clinical criteria and verified by genetic testing
Patients aged between 5 and 15 years old
A score of [e.g.,5 to 17] on the scale for the Assessment and Rating of Ataxia(SARA)
Providing written informed consent by parents or legal guardians
Patients who are medically stable at the time of enrollment
Exclusion criteria;
Presence of other severe progressive neurological disorder
History of or active malignancy
Active uncontrolled infections
History of severe allergy or anaphylaxis to any component used in the cell preparation or infusion process
Unwillingness of the family to comply with study protocol or follow up schedule
Intervention groups
*Treatment with intravenous and intrathecal infusion of allogenic mesenchymal stem cell
*Human bone marrow derived mesenchymal stem cell
*Intravenous and intrathecal infusion at a dose of 1 million cells per kilogram of body weight
*Three infusion, administered one month apart
*premedication with antihistamine 30 minutes prior to infusion
Main outcome variables
*Efficacy: change in scale for the assessment and rating of Ataxia(SARA) score from baseline to 12 months post treatment
*Safety: incidence of treatment related serious adverse events within 12 months
General information
Reason for update
Acronym
CellTX-AT
IRCT registration information
IRCT registration number:IRCT20250528065956N1
Registration date:2025-09-19, 1404/06/28
Registration timing:prospective
Last update:2025-09-19, 1404/06/28
Update count:0
Registration date
2025-09-19, 1404/06/28
Registrant information
Name
Amirali Latifian Alaf
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 51 3841 5352
Email address
latifianaa4021@mums.ac.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2025-10-17, 1404/07/25
Expected recruitment end date
2025-12-16, 1404/09/25
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Evaluation of the safety and efficacy of treatment for Ataxia-telangiectasia using stem cell transplantation in children at Mashhad medical university
Public title
Safety and Efficacy of Stem cell Transplantation in Patients with Ataxia-telangiectasia
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
The age of onset is between 5 and 15 years old
Informed consent from parents or legal guardians
The definitive diagnosis of the disease is based on clinical and genetic criteria
Exclusion criteria:
Presence of other neurodevelopmental disorder
Dissatisfaction with study participation
History of epilepsy or seizure disorder
Adverse reaction to stem cell injection
Age
From 5 years old to 15 years old
Gender
Both
Phase
1
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
Ethics committee of Mashhad University of Medical Science
Street address
no44,16th Qaem, Ahmadabad St.
City
Mashhad
Province
Razavi Khorasan
Postal code
9183889341
Approval date
2025-05-07, 1404/02/17
Ethics committee reference number
IR.MUMS.REC.1404.053
Health conditions studied
1
Description of health condition studied
Ataxia telangiectasia
ICD-10 code
G11.3
ICD-10 code description
Cerebellar ataxia with defective DNA repair
Primary outcomes
1
Description
Primary outcome1: Efficacy : change in SARA score from baseline to 12 months post treatment - Primary outcome2: Safety: Number of patients with treatment related serious adverse events within 12 months
Timepoint
Timepoint for outcome measurement: Baseline(before treatment)-3 months post treatment- 6 months post treatment- 12 months post treatment
Method of measurement
For SARA score: Clinical assessment by neurologist using standardize SARA scale - For adverse events: Monitoring and recording based on CTCAE v5.0 criteria
Secondary outcomes
1
Description
Improved immune function - Improved life function - Improved daily function - Progression-free survival
Timepoint
Baseline(pre treatment)-3months post treatment- 6 months post treatment- 12 months post treatment
Method of measurement
Immunoglobulin levels: ELISA test from venous blood sample - Quality of life: Standard PedsQL questionnaire completed by parents - Daily functioning: Clinical observation and interview by occupational therapist - Disease progression: Clinical assessment by neurologist
Intervention groups
1
Description
Intervention Group: Patients receiving intravenous and intrathecal infusion of allogenic mesenchymal stem cell(3 doses at 1 month intervals) with24hours monitoring post infusion
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Qaem Hospital
Full name of responsible person
Mehran Beiraghi
Street address
No.44,16th Qaem, Ahmadabad St.
City
Mashhad
Province
Razavi Khorasan
Postal code
9183889341
Phone
+98 915 311 6987
Email
aa2.latifian@gmail.com
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Mohsen Tafaghodi
Street address
Ghaem hospital, Ahmad abad Ave
City
mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Phone
+98 51 3801 2469
Email
latifianaa4021@mums.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Mashhad University of Medical Sciences
Proportion provided by this source
100
Public or private sector
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic
Person responsible for general inquiries
Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Amirali Latifian alaf
Position
Resident
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Ghaem hospital, Ahmadabad Ave
City
mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Phone
+98 51 3801 2469
Email
latifianaa4021@mums.ac.ir
Person responsible for scientific inquiries
Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Amirali Latifian Alaf
Position
Resident
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
ahmadabad Ave.
City
mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Phone
+98 51 3801 2469
Email
latifianaa4021@mums.ac.ir
Person responsible for updating data
Contact
Name of organization / entity
mashhad university of medical sciences
Full name of responsible person
amirali latifian alaf
Position
resident
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
ahmad abad
City
mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Phone
+98 51 3801 2469
Email
latifianaa4021@mums.ac.ir
Sharing plan
Deidentified Individual Participant Data Set (IPD)
Yes - There is a plan to make this available
Study Protocol
No - There is not a plan to make this available
Statistical Analysis Plan
Not applicable
Informed Consent Form
Undecided - It is not yet known if there will be a plan to make this available
Clinical Study Report
Yes - There is a plan to make this available
Analytic Code
Not applicable
Data Dictionary
Not applicable
Title and more details about the data/document
Other than identity and contact information
When the data will become available and for how long
Access period starts 6 months after results are published.
To whom data/document is available
Researchers working in academic and scientific institutions
Under which criteria data/document could be used
It is free with attribution.
From where data/document is obtainable
Dr. amirali latifian
What processes are involved for a request to access data/document
The email must be sent to latifianaa4021@mums.ac.ir and if approved, information will be provided within 2 months.