CLINICAL EFFICACY OF NIV AND MODAFINIL ON EXCESSIVE DAYTIME SLEEPINESS: A

INTERVENTION: Trade Name: PROVIGIL ‐ 100 MG COMPRESSE 30 COMPRESSE Pharmaceutical Form: Tablet Route of administration of the placebo: Oral use CONDITION: Myotonic Dystrophy type 1 ; MedDRA version: 20.0 Level: PT Classification code 10068871 Term: Myotonic dystrophy System Organ Class: 10010331 ‐ Congenital, familial and genetic disorders Therapeutic area: Diseases [C] ‐ Musculoskeletal Diseases [C05] PRIMARY OUTCOME: Main Objective: Change from baseline of EDS as assessed by mean Maintenance of Wakefulness Test (MWT) Primary end point(s): Improvement of Excessive Daytime Sleepiness Secondary Objective: Change from baseline of EDS as assessed by subjective sleepiness scales (Epworth Sleepiness; Scale, ESS and Fatigue and Daytime Sleepiness Scale, FDSS); l Change from baseline of EDS as assessed by 1‐week actigraphy; l Change from baseline of fatigue as assessed by the Fatigue Severity Scale (FSS); l Change from baseline of respiratory function (assessed by ABG and PG) at 3 and at 9 months from; treatment; l Change from baseline of distance walked with 6MWT, of TUG and of 9‐peg hole test; l Change from baseline of cognitive tests (TEA, TMT, Stroop, Wisconsin); l Change from baseline of POMS and depression and anxiety scores; l Change from baseline of SF‐36 and INQoLv2 scores; l Improved compliance (assessed by % days used and mean daily usage as indicated by the; ventilators¿ microchip) Timepoint(s) of evaluation of this end point: after 6 months treatment/intervention SECONDARY OUTCOME: Secondary end point(s): Change from baseline of EDS as assessed by subjective sleepiness scales (Epworth Sleepiness; Scale, ESS and Fatigue and Daytime Sleepiness Scale, FDSS); l Change from baseline of EDS as assessed by 1‐week actigraphy; l Change from baseline of fatigue as assessed by the Fatigue Severity Scale (FSS); l Change from baseline of respiratory function (assessed by ABG and PG) at 3 and at 9 months from; treatment; l Change from baseline of distance walked with 6MWT, of TUG and of 9‐peg hole test; l Change from baseline of cognitive tests (TEA, TMT, Stroop, Wisconsin); l Change from baseline of POMS and depression and anxiety scores; l Change from baseline of SF‐36 and INQoLv2 scores; l Improved compliance (assessed by % days used and mean daily usage as indicated by the; ventilators¿ microchip) Timepoint(s) of evaluation of this end point: 6 months after treatment INCLUSION CRITERIA: Molecular diagnosis of DM1, any range of CTG expansion size (= 50) Adult‐onset DM1 Age range 18‐75 Patients who have never tried NIV or patients who have not been using NIV for at least 3 months prior to enrollment Epworth score = 10 Fatigue and Sleepiness (FDSS): positive answers in items 8‐12 (37) Raw MMSE = 20 Patients having EDS according to MSLT = 8 minutes and ESS = 10 or positive scores for EDS on the FDSS scale Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18‐64 years) yes F.1.2.1 Number of subjects for this age range 35 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5