Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome

Clinical Trial

Offered by: Nemours Children's
Location: Delaware Valley

Trial Name

Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome

What is the trial about?

This study is to learn about the safety and effectiveness of the study drug, pitolisant, in comparison to placebo (a tablet that looks like pitolisant but contains no study drug) in treating some of the symptoms of Prader Willi syndrome, such as excessive daytime sleepiness.

Who can participate?

This study is for male or female patients ages 6 to 65 who have been diagnosed with Prader-Willi syndrome and excessive daytime sleepiness (EDS).

What is involved?

This is a study with two parts. The first part is a double-blind period and the second part is an open-label period. During the double-blind period, you (parent/legal guardian), your child’s caregiver, your child’s study doctor and the sponsor will not know if your child is taking the lower dose of the study drug, the higher dose of the study drug or a placebo (a tablet that contains no study drug). During the open-label period of the study, your child will receive pitolisant; you, your child, your child’s study doctor and the sponsor will know the dose that he/she is taking. It is expected that participation in this study for you and your child will last about 17 weeks during the double-blind period and is expected to last multiple years during the open-label extension period. During the double-blind period, the study will include five planned visits at the study clinic and five planned phone calls. During the open-label period, the first four visits at the study clinic will occur every three months, and after that once every six months until the study is finished. There will also be planned phone calls.

Your child will be required to take the study medication every day. During the first part of this study, you will be asked to return to the hospital for up to five study visits and you will be asked to stay at the study clinic overnight with your child for 1-2 nights for two of these visits. During the second part of this study, you will have a study visit once every three months for the first nine months and then every six months for the second and remaining years. As the parent/caregiver you will be required to answer questions about your child’s behavior, hunger and sleepiness.