Ignite Creation Date:
2024-05-06 @ 8:23 PM
Last Modification Date:
2024-10-26 @ 3:26 PM
Study NCT ID:
NCT06366464
Status:
RECRUITING
Last Update Posted:
2024-07-03
First Post:
2023-10-09
Brief Title:
A Study of Pitolisant in Patients With Prader-Willi Syndrome
Sponsor:
Harmony Biosciences LLC
Organization:
Harmony Biosciences LLC
Study Overview
Official Title:
A Phase 3 Randomized Double-Blind Placebo-controlled Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients With Prader-Willi Syndrome
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a Phase 3 randomized double-blind placebo-controlled multicenter global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome
The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness EDS in patients 6 years of age with Prader-Willi syndrome
Secondary objectives include assessing the impact of pitolisant on
Irritable and disruptive behaviors
Hyperphagia
Other behavioral problems including social withdrawal stereotypic behavior hyperactivitynoncompliance and inappropriate speech
The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness EDS in patients 6 years of age with Prader-Willi syndrome
Secondary objectives include assessing the impact of pitolisant on
Irritable and disruptive behaviors
Hyperphagia
Other behavioral problems including social withdrawal stereotypic behavior hyperactivitynoncompliance and inappropriate speech
Detailed Description:
The study will consist of an up to 45-day ScreeningBaseline Period a Double-Blind Treatment Period and an optional Open-Label Extension Period
After completion of all Baseline assessments patients who meet all eligibility criteria will be randomized 11 to receive once daily pitolisant or matching placebo During the Double-Blind Treatment Period in-person visits will be at Day 29 Day 57 and Day 77 Patients who do not elect to enter the Open-Label Extension Period will have follow-up visits 15 days and 30 days after the final dose of study drug
During the optional Open-Label Extension Period in-person visits will be at Day 113 Day 260 and Day 441 Patients will have follow-up visits 15 days and 30 days after the final dose of pitolisant
After completion of all Baseline assessments patients who meet all eligibility criteria will be randomized 11 to receive once daily pitolisant or matching placebo During the Double-Blind Treatment Period in-person visits will be at Day 29 Day 57 and Day 77 Patients who do not elect to enter the Open-Label Extension Period will have follow-up visits 15 days and 30 days after the final dose of study drug
During the optional Open-Label Extension Period in-person visits will be at Day 113 Day 260 and Day 441 Patients will have follow-up visits 15 days and 30 days after the final dose of pitolisant
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None