Clinical Trials Logo

Clinical Trial Summary

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.


Clinical Trial Description

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 30 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 lower dose pitolisant, higher 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04886518
Study type Interventional
Source Harmony Biosciences, LLC
Contact
Status Active, not recruiting
Phase Phase 2
Start date June 28, 2021
Completion date October 2024

See also
  Status Clinical Trial Phase
Recruiting NCT02880735 - Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy N/A
Active, not recruiting NCT06089018 - Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy
Completed NCT05662150 - Low-frequency Repetitive Nerve Stimulation in Myotonic Dystrophy Type 1 N/A
Active, not recruiting NCT04698551 - NIPD on cffDNA for Triplet Repeat Diseases
Completed NCT05027269 - Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients Phase 1/Phase 2
Completed NCT04712422 - Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1
Recruiting NCT05006924 - Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1
Recruiting NCT04656210 - Myotonic Dystrophy - Vascular and Cognition
Completed NCT04634682 - Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1 N/A
Withdrawn NCT06270186 - Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach N/A
Not yet recruiting NCT06300307 - Study of ATX-01 in Participants With DM1 Phase 1/Phase 2
Completed NCT02729597 - Tracking the Brain in Myotonic Dystrophies: a 5-year Longitudinal Follow-up Study N/A
Recruiting NCT06138743 - Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Phase 1
Not yet recruiting NCT05532813 - Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease) Phase 3
Not yet recruiting NCT05865483 - Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)
Completed NCT05036447 - Myotonic Dystrophy Type 1 and Resistance Exercise N/A
Recruiting NCT03424460 - Venous Thromboembolism in Myotonic Dystrophy Type 1 N/A
Completed NCT04001920 - Effects of a Multiple Component Training Program on Muscles in Adults With Myotonic Dystrophy Type 1 N/A
Completed NCT02858908 - Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy Phase 2
Completed NCT04018820 - Effects of a 12-week Strength Training Program in Men With Myotonic Dystrophy Type 1 N/A