Trial studying safety and effects 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 Atipeksen?
Atipeksen is an investigational medicine being studied as a potential treatment for Ataxia Telangiectasia. It is designed to restore normal splicing patterns in the ATM gene for patients with a specific mutation.
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
- The study will see if the therapy is safe and if it can slow or stop the worsening of neurological symptoms in these patients.
- Who it's for
- It is looking for people with Ataxia‑telangiectasia who carry the ATM c.7865C>T mutation.
- What's involved
- Participants will receive the therapy through an injection into the spinal fluid and will have regular medical visits and tests to monitor safety and effectiveness.
- Time commitment
- The trial will last several months, with multiple clinic visits and follow‑up appointments to track progress.
- 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
- People with classic A-T confirmed by genetic testing
- Must have a specific ATM gene change (c.7865C\>T)
- Blood clotting or bleeding problems
- Brain conditions raising pressure inside the head
- Serious heart or breathing problems
- Overall health and stability
See the full eligibility criteria
- INCLUSION/
- People with classic A-T confirmed by genetic testing
- Must have a specific ATM gene change (c.7865C\>T)
- Blood clotting or bleeding problems
- Brain conditions raising pressure inside the head
- Serious heart or breathing problems
- 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 is enrolling
- 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, but 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?
This study is enrolling now at Boston, Massachusetts, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.