Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
Recruiting
Phase 1
Phase 2
Interventional Study
Ataxia Telangiectasia
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 0 – 17
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 10 (estimated)
- Sponsor
- Timothy Yu · Other
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
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. The investigators will conduct a clinical trial to study the safety and efficacy of intrathecal administration of atipeksen, a targeted genetic therapy that restores ATM gene function in A-T individua…
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
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:
* B…
Contacts