Study

The A-T Study at a Glance

Potential Treatment

The Mechanism of Action at a Glance

The potential treatment contains the active ingredient dexamethasone, which has been used to treat other diseases. It is a corticosteroid that is being studied to see whether it may improve the neurological symptoms of A-T.^1,2

In the first step, healthcare professionals draw a small amount of blood from the patient. Using a special sterile technique, the potential treatment places the active ingredient within the extracted red blood cells, which are then administered back to the patient by infusion within two hours of blood draw. In this way, the drug is released from their own patient’s red blood cells over a 3-4 week span. This method reduces the amount of steroids circulating freely in the bloodstream and thus may potentially reduce side effects.

Procedure

How the A-T Study works

Inform & check participation

You can find important information about the A-T Study on this website. Take your time to read through everything. Then, you can use the commitment-free questionnaire to check whether you or your child meet the most important study participation criteria and are likely to be eligible to participate.

Can you take part?

Book an appointment

At the end of the questionnaire, you can leave us your contact details without obligation if you or your child could be suitable based on your information. Our Patient Information Service will then contact you personally in a commitment-free telephone call. We will answer further questions and clarify the next steps with you. We are also happy to provide you with information for your or your child's treating doctor.

First visit to the study site

You (and your child) will meet the study doctor at the first appointment at the study site. The study doctor will provide you with detailed information about the study and check your or your child’s medical history and medication to ensure that all requirements for participation are met. You will receive all the information in written form so that you can read through it at your leisure. Once you have understood everything, have had all your questions answered to your satisfaction and if you decide to participate you can sign the consent form together with the study doctor. Your study doctor will guide you through each step of the process.

Preliminary investigation

After you have given written consent for study participation, preparation for study participation begins with a preliminary examination: Your study doctor will conduct a series of tests to confirm study participation is still appropriate for you or your child.

Treatment phase

During the treatment phase, you or your child will randomly receive a specific dose of either the study drug or the placebo (a dummy drug that looks like the investigational drug but contains no active ingredient) 6 times at intervals of around 28 days. There is, therefore, a 50% chance that you or your child will receive the placebo, not the active ingredient. In both cases, blood is first taken from you or your child, prepared with the (placebo) drug, and then given back to you or them by infusion using the new potential treatment. You will not know whether you or your child have received the study drug or placebo; your study doctor will also not know, but your study doctor can find out in an emergency.

Follow-up & conclusion

At the end of the 6-month treatment phase, there will be a final visit and then a telephone follow-up one month later. If the study is successfully completed, you or your child may be able to voluntarily continue the new potential treatment as part of a study extension. There is no placebo in this study extension, so everyone will receive the study drug. Participation in this extension to the first study is also voluntary, so there is no requirement to continue participation if you do not wish yourself or your child to continue.

Are You Interested in the A-T Study?

Use our commitment-free questionnaire to find out whether you or your child are eligible to participate in the study.

Sources