Status:
NOT_YET_RECRUITING
Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
Lead Sponsor:
Timothy Yu
Conditions:
Ataxia Telangiectasia
Eligibility:
All Genders
Up to 17 years
Phase:
PHASE1
PHASE2
Brief Summary
This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene. ...
Detailed Description
The goal of this protocol is to study the safety and efficacy of the investigational drug atipeksen, a mutation-specific antisense oligonucleotide (ASO), in individuals with ataxia telangiectasia (A-T...
Eligibility Criteria
Inclusion
- INCLUSION/EXCLUSION CRITERIA:
- Who can take part:
- People with classic A-T confirmed by genetic testing
- Must have a specific ATM gene change (c.7865C\>T)
- Must also have another ATM change that causes A-T
- Who cannot take part:
- People with health problems that make lumbar puncture unsafe:
- Blood clotting or bleeding problems
- Brain conditions raising pressure inside the head
- Serious heart or breathing problems
- Infection near the lower back
- Other things doctors will check:
- Overall health and stability
- Any medicines that might cause problems
- Past difficulties with lumbar punctures
- Any other safety concerns
Exclusion
Key Trial Info
Start Date :
November 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2035
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT07215416
Start Date
November 1 2025
End Date
December 1 2035
Last Update
October 10 2025
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