About the Koala Study

The purpose of the Koala study is to evaluate an investigational treatment called NNZ-2591, to see if it can improve how children, 3 to 12 years of age, with Phelan-McDermid Syndrome feel and function.

If the study doctor agrees that this study is a good fit for you and your child and you decide to have them participate, your child will be assigned by chance (like flipping a coin) to receive either NNZ-2591 or a placebo, twice each day for 13 weeks.

A placebo is an inactive treatment that looks and tastes like the investigational treatment, but does not contain any active ingredient.

This is a “double-blinded” study, which means neither you nor the study doctors would know which treatment your child is receiving.

Placebos and blinding are key to confirming that any effects seen in the study are due to NNZ-2591. Your child will be closely monitored by study doctors throughout the treatment period.

Participation in the study will last for a total of about 19 weeks. You will be expected to bring your child to 5 on-site clinic visits. You will also have 6 phone or video calls (your child does not need to attend these).

You can visit clinicaltrials.gov/study/NCT07281079 for more information on the study assessments, entry criteria and site locations. For additional site locations, please contact Neuren’s Medical Information Team at medicalinformation@neurenpharma.com

Optional Participation in the Open-label Extension (OLE) Study
If your child completes all the visits in the Koala study and is eligible, you will have the option to enroll them in a long-term, open-label extension of the study. All participating children in the extension study will receive NNZ-2591 (there is no placebo group), this is known as an “open-label” study. This study is planned to last 52 weeks.

Participating in a clinical study may involve potential benefits as well as potential risks.

You will be provided with details about the study, including potential risks, by the study doctor as part of the assessment to determine if your child qualifies to participate.

The safety of participating children is the top priority of any clinical study.

Your child’s health will be carefully monitored throughout the study. You can choose to withdraw your child from the study at any time and for any reason without affecting their regular medical care.