A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia-Telangiectasia.
This research study is investigating a new treatment called EryDex for people with Ataxia-Telangiectasia, often shortened to A-T. A-T is a rare genetic condition that affects the nervous system and immune system. The main goal is to see if EryDex can improve symptoms relating to the brain and movement, especially in children aged 6 to 9. Participants will receive either EryDex or a placebo (a treatment that looks the same but has no active medicine) in a way that prevents anyone involved from knowing who gets what, ensuring fair results. Researchers will carefully measure changes in symptoms over time to understand if EryDex is truly beneficial. The study is particularly looking for people aged 18 and older, but children aged 6-9 are the focus for the main analysis.
At a glance
What is this study about?
This study is exploring a new treatment called EryDex for a rare genetic condition known as Ataxia-Telangiectasia, or A-T. A-T mainly affects the brain and immune system, causing problems with movement, balance, and coordination. The purpose of this study is to find out if EryDex can help improve these symptoms, especially those related to the brain and movement.
Researchers will compare EryDex to a 'placebo', which is a substance that looks exactly like the treatment but contains no active medicine. This helps scientists truly understand if any improvements are due to the treatment itself or other factors. The study is 'double-blind', meaning neither the participants, their families, nor the study doctors will know who is receiving EryDex and who is receiving the placebo. This setup is crucial for ensuring the study results are as unbiased and accurate as possible.
The main focus of the study is to measure how well EryDex improves symptoms related to the brain and movement, particularly in children aged 6 to 9. They will use a special scoring system to track changes over time. They will also look at overall improvements in daily life and well-being. This type of study, called a Phase III trial, is a very important step in determining if a new treatment should become widely available.
Key takeaways
- This study is testing a new treatment, EryDex, for Ataxia-Telangiectasia (A-T).
- It aims to see if EryDex can improve brain and movement symptoms, especially in children aged 6-9.
- Participants will receive either EryDex or a dummy treatment (placebo).
- It's a 'double-blind' study, meaning no one knows who gets which treatment.
- The study involves regular clinic visits and health assessments over several months.
Who may be eligible?
This study is open to both men and women. While the research team is looking for participants of all adult ages, the main part of the study focused on children will look specifically at those between 6 and 9 years old.
If you are an adult aged 18 or over and have Ataxia-Telangiectasia, you might be able to take part. The study also has a special focus on children with A-T who are between 6 and 9 years old. This means that if you or your child falls into these age groups and has A-T, you could be eligible to join.
There will be other detailed health checks to make sure the study is safe and right for you, but these are the main age and condition requirements.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Do you (or your child) have Ataxia-Telangiectasia (A-T)?
- Are you 18 years old or older?
- If it's for a child, are they aged between 6 and 9 years old?
- Are you able to attend regular clinic appointments for the study?
What does participation involve?
Taking part in this study would involve regular visits to the clinic over a number of months. During these visits, medical staff will carry out various assessments to monitor your health and how your symptoms are changing. You will be given either the study drug, EryDex, or a placebo (a dummy treatment). Neither you nor your doctors will know which one you are receiving. The study will track how your symptoms change from the beginning until around 6 months (Day 168) after starting the treatment. There will also be follow-up appointments to ensure your safety and monitor any long-term effects. The full duration of your involvement, including all visits and follow-up, will be clearly explained by the study team.
Potential risks and benefits
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Common questions
What is Ataxia-Telangiectasia (A-T)?
A-T is a rare genetic condition that mainly affects the brain's ability to control movement and balance, and it can also weaken the immune system.
What is a 'placebo'?
A placebo is a dummy treatment that looks exactly like the real medicine but contains no active ingredients. It helps researchers know if the new drug is truly effective.
What does 'double-blind' mean?
It means that neither the patient nor the doctor knows whether the patient is receiving the study drug or the placebo. This keeps results fair and unbiased.
Will I get the actual treatment?
You will have a 50/50 chance of receiving either the EryDex treatment or the placebo. The study is set up this way to ensure accurate results.
How long does the study last?
The main treatment period is about 6 months (168 days), followed by additional monitoring. The full commitment will be detailed by the study team.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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