This study is looking at a condition called narcolepsy type 1, which is a sleep disorder where people experience excessive daytime sleepiness and sudden muscle weakness triggered by emotions, a symptom known as cataplexy. The study will use a medication called TAK-861, also known as oveporexton, which comes in tablet form and is taken by mouth. This medication has been designated as an orphan drug, meaning it is being developed to treat a rare condition.
The purpose of this study is to understand how the body of children and teenagers processes this medication by measuring drug levels in the blood over time, and to check if the medication is safe and acceptable for young people to take. The study will include children from 6 years old up to 18 years old who have been diagnosed with narcolepsy type 1. Participants will need to have either a specific genetic marker or very low levels of a brain substance called hypocretin in their spinal fluid, which are both signs of this type of narcolepsy. They will also need to have a body mass index within a certain range and be in generally good health as determined by various medical tests including blood work, physical examination, heart rhythm recording, and vital signs measurements.
During the study, participants will receive a single dose of the medication, and doctors will collect blood samples at different times to see how the drug moves through the body. The study will measure things like the highest amount of drug in the blood, how long it takes to reach that level, and how long the drug stays in the body. Researchers will also watch for any unwanted effects and ask participants and their parents or guardians about how acceptable the medication is to take and what it tastes like. This is an open-label study, which means both the doctors and participants will know that everyone is receiving the active medication rather than a placebo.
Italy 
