A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label Extension
The primary objective of this study is to evaluate the safety and efficacy of pitolisant
compared with placebo in treating excessive daytime sleepiness (EDS) in patients with
Myotonic Dystrophy Type 1 ages 18 to 65 years.
The secondary objectives of this study are to assess the impact of pitolisant on fatigue,
cognitive function and the burden of disease along with assessing the long-term safety and
effectiveness of pitolisant in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
The study will consist of a Screening Period, an 11-week Double-Blind Treatment Phase
(including a 3-week Titration Period and an 8-week Stable Dose Period), and an optional Open
Label Extension (OLE) Phase. The OLE Phase will last approximately one year for each patient
or until the Sponsor elects to terminate the study.
Approximately 78 patients ages 18 to 65 years who meet all eligibility criteria will be
randomized at the Baseline Visit in a 1:1:1 ratio to low dose pitolisant, high dose
pitolisant, or matching placebo. In the Double-Blind Treatment Phase, patients will be
titrated to their randomized stable dose of study drug during the 3-week Titration Period.
After completion of the 3-week Titration Period, patients will continue to take study drug at
their randomized stable dose once daily in the morning upon wakening for an additional 8
weeks of blinded treatment (Stable Dose Period). The duration of the Double-Blind Treatment
Phase will be 11 weeks.
Following the 11-week Double-Blind Treatment Phase, eligible patients will be given the
opportunity to participate in an optional OLE Phase. During the OLE Phase, all eligible
patients will receive treatment with open-label pitolisant. Patients will first undergo a
3-week Titration Period to a maximum target dose, after which they will continue to take
their dose of pitolisant once daily in the morning upon wakening until the end of the study.
The patient's dose of pitolisant may be adjusted during the OLE phase.
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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