FAQs

SLC6A1-NDD is a rare disorder, so there isn’t a lot of information available how the disorder presents and progresses over time. In order to develop new ways to help people living with the disorder, more data is needed. With the SPIRIT Study we want to help close that gap and better understand how SLC6A1-NDD develops over time.

If your loved one joins the study, here are some of the things you will be asked to do:

• Attend study visits: The study includes a total of 7 visits over a period of 2 years.
• Have tests and assessments: These include physical exams, vital signs (like blood pressure and heart rate), developmental assessments, a cheek swab for genetic testing and electroencephalogram (EEG) to monitor brain activity. There is also one optional blood draw for laboratory analysis.
• Fill in a daily seizure diary: The study team will provide this to you and teach you how to use it.
• Complete questionnaires on your loved one’s health and well-being by using a tablet device (can be done at home) and agree to 6 phone interviews by trained healthcare professionals about your loved one’s behavior.

If you allow your loved one to join the SPIRIT Study, their participation will last approximately 2 years with a total of 7 study visits.

If you decide that you no longer want to take part, you and your loved one can leave the study at any time for any reason.

The SPIRIT Study is an observational study only so no study medication will be given.

Study participants can continue on their usual medications that they were already taking when they joined the study. The use of 4-phenylbutyrate if taken at a stable dose will be permitted. It will be useful to keep track of any changes to your loved one’s medications or therapies during the study so that this can be documented.

There are risks and discomforts in most studies, and these include risks associated with the procedures done as part of study visits. Your study team will inform you in detail about potential risks and benefits before you decide to join the study. Some known potential risks and discomforts in this study are:

• Taking blood pressure by using an inflatable cuff may cause mild discomfort in your loved one’s arm.
• The electroencephalogram (EEG) may cause discomfort or skin irritation/redness where the electrodes are placed on your loved one’s head.
• Having a cheek swab can cause mild irritation or soreness in the mouth.
• Having blood samples taken (optional) may cause pain or bruising

Joining in a clinical study is a very personal and individual decision, and you should consider whether this is the right choice for you and your loved ones. You can stop participation at any time without giving any reason. Deciding not to take part will not affect your loved one’s usual medical care now or in the future.

You will not receive any payment for participation in the SPIRIT Study, but reasonable travel or meal expenses can be reimbursed. Please speak to your study team about how this works. You will not have to pay for any study-related tests and assessments.

The SPIRIT Study is sponsored by UCB, a global biopharma company based in Belgium that is focusing on neurology and immunology disorders.

All personal data collected for the study that can identify your loved one will be “coded” to remove any part of the personal data that could identify them. Using a code to identify your loved one helps researchers keep their information confidential.

No. You can contact the study center to find out more about participating without any commitment. Contacting a study center does not mean that you and your loved one must or will be able to join the study.