Things You Should Know / Most common Questions About the Study/ FAQ

Clinical research studies (or ‘trials’) look for new ways to develop potential treatment options. This may involve comparing investigational treatments (treatments that have not been approved by a regulatory body) to “comparator” medicines (i.e. an approved medicine) or inactive treatments (i.e. a placebo), by splitting participants into groups and randomly assigning them to one of the treatments.

Even if participants don’t directly benefit from the results of the clinical study, the information collected adds to scientific knowledge and may help families like yours in the future.

Caregivers and their children who take part in clinical studies are essential to the process of improving pediatric medical care.

Participation in the study will last for a total of about 19 weeks.

If your child completes all the visits in the Koala study and is eligible, you may have the option to enroll them in an open-label extension of the study. All participating children in the open-label extension study will receive NNZ-2591 (there is no placebo group). This study is planned to last 52 weeks.

Informed consent is a process in clinical research studies where the participant or their caregiver is provided with the details about the study, including any potential risks, before making their decision for them or their child to join the study. Participants or their caregivers are required to sign an ‘informed consent form’ before beginning any of the study activities. This is not a contract, and participants can leave the study at any time and for any reason.

The study drug, study-related procedures and travel associated with clinic visits are provided at no cost.

All the potential risks and benefits of the study will be explained to you in detail before you decide if you want your child to take part. There are potential risks associated with taking any medical treatment, including the study drug. Your child’s symptoms may get better, stay the same, or get worse. If you choose to have your child join the study, your study doctor will review the potential risks with you.

There are also potential risks associated with the procedures and tests your child will have during the study. The assessments performed as part of the study may cause your child tiredness, frustration and may be a source of behaviors that are not helpful or are potentially harmful.

If your child completes all visits in the Koala study and is eligible, you may have the option to enroll them in an open-label extension of the study. All participating children in the open-label extension study will receive NNZ-2591 (there is no placebo group). This is planned to last for 52 weeks.

You can stop your child from taking part in this study at any time and for any reason. You do not need to give a reason. If you decide you do not want your child to take part, or if you decide you want your child to stop being in the study, this will not affect the normal treatment your child receives, there will not be a penalty, and your child will not lose any benefits they would otherwise have.

If you decide to take your child out of the study early, you may be asked to attend additional study visits so the study doctor can assess their health.