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IRCT20250528065956N1 IRCT 2025-09-19 Mashhad University of Medical Sciences Safety and Efficacy of Stem cell Transplantation in Patients with Ataxia-telangiectasia CellTX-AT Evaluation of the safety and efficacy of treatment for Ataxia-telangiectasia using stem cell transplantation in children at Mashhad medical university 2025-10-17 anticipated 5 Complete https://irct.ir/trial/86175 interventional Randomization: N/A, Blinding: Not blinded, Placebo: Not used, Assignment: Single, Purpose: Treatment. 1 Ataxia telangiectasia. Intervention Group: Patients receiving intravenous and intrathecal infusion of allogenic mesenchymal stem cell(3 doses at 1 month intervals) with24hours monitoring post infusion. Yes - There is a plan to make this available What will be shared: Other than identity and contact information When: Access period starts 6 months after results are published. To whom: Researchers working in academic and scientific institutions Conditions: It is free with attribution. Where to obtain: Dr. amirali latifian How to obtain: The email must be sent to latifianaa4021@mums.ac.ir and if approved, information will be provided within 2 months. Comments:
public Amirali Latifian alaf
Ghaem hospital, Ahmadabad Ave
mashhad Iran (Islamic Republic of) 99199-91766 +98 51 3801 2469 latifianaa4021@mums.ac.ir Mashhad University of Medical Sciences scientific Amirali Latifian Alaf
ahmadabad Ave.
mashhad Iran (Islamic Republic of) 99199-91766 +98 51 3801 2469 latifianaa4021@mums.ac.ir Mashhad University of Medical Sciences Iran (Islamic Republic of) 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 5 years 15 years Both Presence of other neurodevelopmental disorder Dissatisfaction with study participation History of epilepsy or seizure disorder Adverse reaction to stem cell injection G11.3 Cerebellar ataxia with defective DNA repair Treatment - Drugs Intervention Group: Patients receiving intravenous and intrathecal infusion of allogenic mesenchymal stem cell(3 doses at 1 month intervals) with24hours monitoring post infusion 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. 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. Mashhad University of Medical Sciences Approved 2025-05-07 Ethics committee of Mashhad University of Medical Science no44,16th Qaem, Ahmadabad St. Mashhad Razavi Khorasan Iran (Islamic Republic of)