Safety and Efficacy of Stem cell Transplantation in Patients with Ataxia-telangiectasia

Evaluation of the safety and efficacy of treatment for Ataxia-telangiectasia using stem cell transplantation in children at Mashhad medical university

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

IRCT

Registry ID

IRCT20250528065956N1

Enrollment

Registered

2025-09-19

Start date

2025-10-17

Completion date

Unknown

Last updated

2025-10-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Ataxia telangiectasia. Cerebellar ataxia with defective DNA repair

Interventions

Intervention Group: Patients receiving intravenous and intrathecal infusion of allogenic mesenchymal stem cell(3 doses at 1 month intervals) with24hours monitoring post infusion.

Eligibility

Sex/Gender

All

Age

5 Years to 15 Years

Inclusion 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

Exclusion criteria: Presence of other neurodevelopmental disorder Dissatisfaction with study participation History of epilepsy or seizure disorder Adverse reaction to stem cell injection

Design outcomes

Primary

Measure	Time frame
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.	—

Measure

Time frame

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.

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Secondary

Measure	Time frame
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.	—

Measure

Time frame

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.

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Countries

Iran (Islamic Republic of)

Contacts

Public ContactAmirali Latifian alaf

Mashhad University of Medical Sciences

latifianaa4021@mums.ac.ir+98 51 3801 2469

Outcome results

None listed

Source: IRCT (via WHO ICTRP) · Data processed: Feb 4, 2026