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Tests treatment safety and results for Ataxia Telangiectasia
Official title Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
ClinicalTrials.gov ID: NCT07215416
What this study is testing
What is Antisense oligonucleotide targeting the ATM gene?
Antisense oligonucleotide targeting the ATM gene is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for ataxia telangiectasia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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 individuals bearing the recurrent ATM c.7865C\>T variant.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 17
You may be able to join if
- INCLUSION/
You likely can't join if
- 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
See the full eligibility criteria
- INCLUSION/
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.