A Study of Pitolisant in Patients With Prader-Willi Syndrome
This study is looking at a new medication, Pitolisant, for individuals aged 6 and above who have Prader-Willi syndrome. We want to find out if Pitolisant can help reduce feeling too sleepy during the day and improve some challenging behaviors like irritability, constant hunger, and social difficulties. Participants will receive either Pitolisant or a placebo (a dummy pill) for a set period, and neither they nor their doctors will know which one they are taking. This helps us accurately measure the medication's effects. The study also tracks participants afterwards to understand any longer-term effects. This research aims to find better ways to manage life with Prader-Willi syndrome.
At a glance
What is this study about?
This research study is called a 'clinical trial' and it's looking at a medicine called Pitolisant. It's for people aged 6 and older who have Prader-Willi syndrome. The main goal is to see if Pitolisant can help people feel less sleepy during the day. Many people with Prader-Willi syndrome experience 'excessive daytime sleepiness,' which means they feel very tired and want to sleep a lot, even after a full night's rest. This can make everyday activities difficult.
Beyond sleepiness, the study also wants to understand if Pitolisant can help with other common challenges in Prader-Willi syndrome. These include things like feeling irritable, having disruptive behaviors, constant hunger (called hyperphagia), withdrawing from social situations, repetitive actions, being very active, not wanting to follow instructions, or using inappropriate speech. By studying these areas, researchers hope to find out if Pitolisant could improve the general well-being and daily lives of people living with Prader-Willi syndrome.
This kind of study is carefully planned to make sure we get reliable answers. It's a 'Phase 3' study, which means the medication has already been tested in smaller groups, and now we're looking at a larger group of people. Participants will receive either the actual medicine or a 'placebo,' which looks the same but contains no active drug. This is done so we can clearly see if any changes are due to the medicine itself or other factors. Both the patients and their doctors won't know who is getting which, a method called 'double-blind.'
Key takeaways
- This study is for people with Prader-Willi syndrome who are aged 6 or older.
- It tests Pitolisant to see if it helps with excessive daytime sleepiness and other behaviors.
- Participants will receive either Pitolisant or a placebo (dummy pill).
- The study involves several in-person visits and a possible extended treatment period.
- A consistent caregiver is needed to help with study tasks.
- You can stop participating at any time.
Who may be eligible?
To join this study, people need to have a confirmed diagnosis of Prader-Willi syndrome through a genetic test. They must also experience excessive sleepiness during the day. It's very important that there's a parent or caregiver, preferably the same person throughout, who can help with the study tasks and assessments.
The study also needs to be sure that the patient, their parents, or caregivers understand what's involved and can follow the study instructions, including how to take the medicine by mouth.
However, some things might prevent someone from joining. For example, if someone has sleep apnea (a condition where breathing stops and starts during sleep) that isn't properly managed, they can't take part. Also, if their sleepiness is caused by a different sleep problem or medical condition, they wouldn't be suitable. Finally, if they've recently taken part in another study involving a new medication or treatment within the last month or so, they would also not be eligible.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Do you have a genetically confirmed diagnosis of Prader-Willi syndrome?
- Do you experience a lot of sleepiness during the day?
- Do you have a parent or caregiver who can help you with the study?
- Is your sleep apnea (if you have it) well-controlled?
- Have you avoided other experimental medications or treatments in the last month?
What does participation involve?
If you decide to join this study, your journey begins with a 'Screening/Baseline' period that can last up to 45 days. During this time, the research team will do tests and ask questions to make sure you're a good fit for the study. Once all checks are complete and you meet the requirements, you'll be assigned either Pitolisant or a matching dummy pill (placebo).
During the main treatment part of the study, which is 'double-blind' (meaning neither you nor your doctor knows if you're taking the real medicine or the placebo), you'll have in-person visits at the clinic on roughly Day 29, Day 57, and Day 77. These visits are important for monitoring your health and checking how you're responding to the treatment. If you choose not to continue into the next phase, there will be two follow-up phone calls or visits about 15 and 30 days after your last dose of the study medicine.
After the main treatment period, there's an optional 'Open-Label Extension' period. This means everyone in this part will receive Pitolisant, and everyone will know they are getting the active medicine. If you choose to continue, you'll have more in-person visits around Day 113, Day 260, and Day 441. Again, after your final dose of Pitolisant in this extended period, there will be two follow-up checks about 15 and 30 days later. The total duration of participation depends on whether you join the optional extension.
Potential risks and benefits
Locations (54)
- Santa Monica Clinical TrialsVerified postcodeLos Angeles, United States· Recruiting
- Center of Excellence in Diabetes and EndocrinologyVerified postcodeSacramento, United States· Recruiting
- Rady Children's Hospital - Scan DiegoVerified postcodeSan Diego, United States· Recruiting
- Colorado Children's HospitalVerified postcodeAurora, United States· Recruiting
- Nemours Children's HospitalVerified postcodeWilmington, United States· Recruiting
- Atlanta Diabetes AssociatesVerified postcodeAtlanta, United States· Withdrawn
- Emory University School of MedicineVerified postcodeAtlanta, United States· Recruiting
- Rare Disease ResearchVerified postcodeAtlanta, United States· Withdrawn
- Ann And Robert H Lurie Children's Hospital of ChicagoVerified postcodeChicago, United States· Recruiting
- Riley Children's HospitalVerified postcodeIndianapolis, United States· Recruiting
- Johns Hopkins HospitalVerified postcodeBaltimore, United States· Recruiting
- Mayo Clinic-PPDSVerified postcodeRochester, United States· Recruiting
Common questions
What is a 'placebo'?
A placebo is a 'dummy' pill that looks just like the study medication but doesn't contain any active drug. It helps researchers see if the real medicine is truly effective.
What does 'double-blind' mean?
Double-blind means that neither you, your family, nor your study doctor will know whether you are taking Pitolisant or the placebo. This helps ensure fair and accurate results.
Will I have to pay to be in the study?
Clinical trials generally cover the cost of study-related medication and visits. You should discuss any potential costs with the study team.
What is 'excessive daytime sleepiness' (EDS)?
EDS means feeling very tired and sleepy during the day, even after getting enough sleep at night. It can make it hard to stay awake and focus.
What is an 'Open-Label Extension Period'?
This is an optional longer period after the main study where all participants receive the active study drug (Pitolisant) and everyone knows they are receiving it. It helps gather more information on longer-term effects.
How to find out more
Ann Adee
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.