PHASE 3 STUDY
About the Koala Study
The Koala study is evaluating an investigational treatment called NNZ-2591, to see if it can improve how children 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 which looks 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.
We understand that using placebos and blinding can cause some uncertainty, but they are key to confirming that any effects seen in the study are due to the potential new medicine. 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).
If your child completes all the visits in the Koala study and are eligible, you will have the option to enroll them in a long-term extension of the study. All participating children in the extension study will receive NNZ-2591 (there is no placebo group) This study is planned to last 52 weeks but could be extended to allow continued access to NNZ-2591.
You can visit clinicaltrials.gov/study/NCT05025241 for more information on the study assessments, entry criteria and site locations.
Your Child's Safety
The safety of participating children is the top priority of any clinical study. You will be provided with all the details about the study, including any potential risks, before deciding to have your child join the study. Your child’s health will be carefully monitored throughout the study to ensure their safety. You can choose to withdraw your child from the study at any time and for any reason without affecting their regular medical care.
See if your child may qualify
To determine if your child is eligible for this study, you will be asked some personal questions about your child’s health and current condition.
You can stop answering questions at any time and you don’t have to answer any questions that make you feel uncomfortable.
The information you provide is confidential and will never be sold or shared with outside parties. Feel free to view our privacy policy.