Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

Inclusion criteria

• ✓. Is able to provide voluntary, written informed consent.
• ✓. Has a diagnosis of DM1 confirmed by genetic testing (cytosine-thymine-guanine \[CTG\] repeat of ≥100) from the Screening Visit.
• ✓. Male or female patients ages 18 to 65 years at the time of enrollment.
• ✓. Has a Clinical Global Impression of Severity (CGI-S) assessment of moderate or severe for overall severity of EDS at Screening.
• ✓. If on a wake-promoting treatment that could affect EDS (including stimulants, modafinil, and armodafinil):
• ✓. Must be on a stable dose for at least 2 months prior to Screening and agree to continue the stable dose for the duration of the Double-Blind Treatment Phase of the study (dose adjustments will be permitted in the OLE Phase).
• ✓. If not on a stable dose for 2 months prior to Screening, washout for 5 half-lives prior to randomization and agree to remain off these treatments for the duration of the Double-Blind Treatment Phase of the study.
• ✓. Washout of cannabidiol and tetrahydrocannabinol for 28 days prior to randomization and agree to remain off for the duration of the Double-Blind Treatment Phase of the study.

Exclusion criteria