Clinical Study for People with A-T

Ataxia-Telangiectasia (A-T): Developing a Potential Treatment for People with A-T

If you or your child have been diagnosed with Ataxia-Telangiectasia (A-T), this study may be of interest to you. You can use our online questionnaire to find out quickly and without obligation, whether participation is possible for you or your child.

Dear patients and parents,

your or your child’s Ataxia-Telangiectasia (A-T) diagnosis can be emotionally and physically draining to a family. Despite medical research efforts, at present there are no approved treatments for A-T. Current treatment regimens focus on alleviating symptoms and delaying progression as best as possible.

Development of a new potential treatmentMedical research is striving to develop treatments that could potentially treat the neurological symptoms of people diagnosed with A-T. In this international phase III clinical trial, “NEAT” by Quince Therapeutics, a potential treatment for A-T is being investigated. The EryDex system aims to potentially enable the safe and long-term administration of an anti-inflammatory agent. Thus, the new approach could potentially lead to improved neurological symptoms.

Patients with A-T can now participateIf you or your child have been diagnosed with A-T and have not participated in a previous A-T trial with EryDex treatment, then this study could be of interest to you. You can find out more about the potential treatment and the study on our website. You can use our questionnaire without obligation to see if participation would be possible for you or your child (further checks to determine suitability will be required by the study doctor).

Thank you very much for your interest in the A-T Study.

Andrew Prayle
Coordinating Investigator of the A-T Study

"The potential treatment is based on an active substance that has already been proven to be effective in other diseases and could potentially lead to improving the symptoms of A-T."

Andrew Prayle

Coordinating Investigator of the A-T Study. Clinical Associate Professor in Paediatric Respiratory Medicine, University of Nottingham UK

Sponsor

Conducted by

Logo Quince Therapeutics

Opportunities

6 Reasons to Participate in the Study

The A-T Study aims to find out whether the potential treatment works for treating the neurological symptoms of A-T.

Opportunities​

6 Reasons to Participate in the Study​

The A-T Study aims to find out whether the potential treatment works for treating the neurological symptoms of A-T.

You or your child might have the opportunity to participate in a study to investigate a potential treatment.

The potential treatment may possibly alleviate neurological symptoms.

The study’s sponsor will cover the study-related treatments and travel expenses.

You or your child will be closely monitored by doctors, nurses and study staff during the study.

By taking part in the study, you or your child may help medical research to potentially develop treatments for A-T that could benefit people with A-T in the future.

You or your child have the opportunity to continue the potential treatment in a 1-year extension study after original study completion.

Possible Risks

As with all clinical trials, participation may be associated with potential risks. The potential treatment may not work or not work as desired, or unexpected events may occur. For this reason, close monitoring is carried out.

Criteria

Are You or Your Child Eligible for the Study?

The A-T Study is mainly aimed at children or young adults diagnosed with Ataxia-Telangiectasia.

You or your child may be eligible to participate in the study if:

Participation in the study is not possible if:​

Participation

How You or Your Child could Potentially Take Part in the Study

Step

1

Get information

Find out more about the details of the A-T Study on our study website. Answers to the most important questions about the study can be found here.

Step

2

Fill out the questionnaire

A questionnaire will tell you whether you or your child may be able to take part.  Afterwards, our Patient Information Service will clarify the next steps with you.

Step

3

Make an appointment

If you are interested in your or your child’s participation, talk to a study doctor commitment-free. Only after your appointment at the study site, can you decide whether you or your child should participate. The decision to not participate will not affect any medical care you or your child receives.

At a Glance

What Will I or My Child Face During the Study Phase?

You will not incur any costs by participating in the study. Read more about the study here.

3

Telephone Appointments

are planned.

8

Visits

to the study site.

8

Months

of participation.

6

Infusions

are part of the treatment.

Study Sites

Here is Where the A-T Study Takes Place

4

Locations

106

Participants

Procedure

This is how the A-T Study works in detail

Contact

Are there any unanswered questions? Get in touch!

Eligibility

Can you or your child take part in the A-T Study?

FAQ

Frequently Asked Questions

EryDex is an innovative system. What makes EryDex unique is that the system loads a corticosteroid into the patient’s own red blood cells before returning their blood back into the patient’s body via infusion. The advantage of this is that the active ingredient is released slowly and directly into the tissue over a long period of time. This reduces the amount of steroid circulating freely in the blood and thus may potentially lead to fewer side effects.1,2

EryDex is an investigational treatment and is not approved for any indication in any region yet.

Yes, previous studies in A-T suggest that agents such as steroids may help patients with Ataxia-Telangiectasia.2,3 A-T symptoms are assessed using specific neurological scales that measure the effects of A-T on patients’ posture, gait, movements (including the eyes) and speech. The corticosteroid used is already approved for the treatment of various long-term inflammatory conditions but not specifically for Ataxia-Telangiectasia.

Throughout the study, you or your child are permitted to continue specific medications, while others will be prohibited so they will not interfere with the study results. If you or your child need to take other medicines during the study, please inform the study doctor or your family doctor that you or your child would like to participate in the A-T Study. Ask them to clarify with the study doctor which prescriptions may be continued during the study.

You must also inform the study doctor about all other studies in which you or your child are participating. If you or your child are taking part in another study, you cannot take part in the A-T Study.

Yes. People participating in the A-T Study are randomly assigned to one of two groups in equal numbers (like tossing a coin): One group receives treatment with an active ingredient, and the other group receives the treatment with a placebo. Both are administered as infusions.

Neither you, your child, the study doctor, nor any other person involved in the study will be informed about which ingredient (active or placebo) you or your child receives. This principle is called “blinding”.

In certain situations, such as a medical emergency, so-called unblinding can take place. This means that your study doctor or the study team can then find out whether you or your child have received the active substance or the placebo.

Upon study completion and if you or your child are willing to continue treatment for another year and the study doctor considers you or your child eligible to continue treatment there will be an opportunity to continue in an additional study where everyone receives the active substance.

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.