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New Autism Research
on the Horizon

The AuRoRa Borealis Study is examining an investigational drug that may help adolescents and adults (ages 15 to 45) with the core characteristics of Autism.

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ABOUT AUTISM

About Autism

Autism is a developmental condition made up of related neurological issues that are intertwined. While different people with Autism experience different characteristics, there are two core characteristics:

Difficulties with social interaction and communication

Restricted and repetitive behaviors

The AuRoRa Borealis Study is evaluating an investigational drug designed to target a neurotransmitter receptor in the brain (i.e., a molecule that is needed to exchange information between cells of the brain) that may help with these core characteristics of Autism.

ABOUT THE STUDY

A clinical research study focused on social and communication difficulties and restricted and repetitive behaviors.

The AuRoRa Borealis Study is evaluating the safety and efficacy of an investigational drug in adolescents and adults (ages 15 to 45) with moderate to severe Autism, which is approximately equivalent to level 2 or 3 support needs.

About the Investigational Drug

While the exact cause of Autism is unknown, researchers believe there may be a link between Autism and changes in levels of a natural substance known as GABA (gamma-aminobutyric acid), which is used by cells in the brain to communicate with each other. The investigational drug is thought to increase the activity of GABA by attaching to specific brain receptors, which researchers believe may be helpful in addressing some of the core characteristics of Autism. The investigational drug is given in the form of tablets, which are taken once a day with water or food.

Study Design

Note: This study requires that the participant has a study support person also participating in the study with them. This person does not have to be the participant’s primary caregiver, but it should be someone the participant knows well, regularly interacts with, and available to support the participant throughout the study duration.

The AuRoRa Borealis Study will compare the investigational drug to a placebo. A placebo is an inactive material, such as a sugar pill, that looks like the investigational drug but does not contain any active medication. Placebos play an important role in clinical research studies, as they help researchers determine if an investigational drug works better or is safer than taking nothing.

Study participants in the AuRoRa Borealis Study will be randomly assigned (like flipping a coin) to one of three study dosing groups:

Low-dose
investigational drug group

High-dose
investigational drug group

Two doses of the investigational drug are tested to see which one, if any, has the most potential to help people with Autism. Neither the study participant, their study support person, nor the study doctor will know who is receiving the investigational drug or placebo; however, the study doctor can find out this information if they feel it is necessary for the participant’s health.

For additional information about the study drugs, please speak with a member of the study staff.

Study Duration: 24 Weeks

Screening / Baseline Period
(4–6 weeks):

You / the participant and study support person will find out if they are fully eligible for the study and decide if they want to take part.

Study Treatment Period
(12 weeks):

You / the participant will take your / their assigned study drug and take part in tests and assessments. Visits will take place on Day 1 and in Weeks 2, 6, and 12. The study team will also call to check on the your / participant’s health and ask a few questions.

Follow-up Period
(6 weeks):

Two after-treatment visits around two and six weeks after the participant took the last tablet for tests and assessments.

ABOUT CLINICAL RESEARCH

A clinical research study is a scientific investigation designed to answer important questions about an investigational drug, such as:

Is it safe?

Does it work?

Which dose may work best?

Are there any side effects?

This is a Phase 2 study, which means it has already been initially tested for safety. In this study, researchers will further evaluate the safety and potential efficacy of the study drug at reducing the core characteristics of Autism. All study drugs must be tested in clinical research studies before they are approved and available to be prescribed by doctors.

Clinical research studies are also monitored by an independent Institutional Review Board (IRB) or Ethics Committee (EC), composed of physicians and experts who are not participating in the study and who are responsible for protecting the safety and rights of participants.

Choosing to take part in a clinical research study is a big decision.

The study team wants to make sure that participants and their study support person is comfortable with all aspects of participating in a clinical research study. The team will speak with participants and study support persons to go over a document called an Informed Consent Form, which will help them understand what the study is about, the potential risks and benefits, and what will be involved before they agree to participate. If a participant and study support person are interested in being part of the AuRoRa Borealis Study, they will be asked to first read and sign the Informed Consent and Assent Forms.

This form contains information about the AuRoRa Borealis Study, including a summary of what the study participant and the study support person may expect during the study, potential risks and benefits, the rights of study participants, and other options that may be available to them. If the participant needs help understanding the study and what is involved, the study team will have pictorial resources to assist them.

Participating in the AuRoRa Borealis Study is completely voluntary. The participant or study support person may choose to stop participating at any time and for any reason.