Clinical trial on the efficacy of non invasive ventilation and modafinil on excessive daytime sleepiness in Myotonic Dystrophy type 1

CLINICAL EFFICACY OF NIV AND MODAFINIL ON EXCESSIVE DAYTIME SLEEPINESS: A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL IN DM1 - ModaNIV

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-000601-36-IT

Enrollment

Registered

2021-06-08

Start date

2016-07-26

Completion date

Unknown

Last updated

2021-08-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

Trade Name: PROVIGIL - 100 MG COMPRESSE 30 COMPRESSE Pharmaceutical Form: Tablet Route of administration of the placebo: Oral use

Eligibility

Sex/Gender

All

Inclusion criteria

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

Exclusion criteria

Exclusion criteria: (i) Hypersensibility to modafinil (ii) Pregnancy or women who are in child bearing age who do not adhere to the guidelines applied during clinical trials “Recomandations related to contraception and pregnancy testing in clinical trials". (iii) Patients who receive drugs that may interfere with modafinil (tricyclic antidepressants like clomipramine, desimipramine, pregabalin, carbamazepine, valproate, lithium, hydantoins, warfarin, propranol, MAO inhibitors, erythromycin, ketoconazole, phenobarbital, rifampin, diazepam, hydantoins) (iv) Cognitive impairment as assessed by interviews or MMSE = 20 (v) Patients with uncontrolled hypertension will be excluded (vi) Patients with cardiac arrhythmias (tachycardia, atrial fibrillation, atrial flutter, bradycardia meaning <60 bpm), ventricular fibrillation) will be excluded (vii) History of seizure

Design outcomes

Primary

Measure	Time frame
Main Objective: Change from baseline of EDS as assessed by mean Maintenance of Wakefulness Test (MWT);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);Primary end point(s): Improvement of Excessive Daytime Sleepiness;Timepoint(s) of evaluation of this end point: after 6 months treatment/intervention	—

Measure

Time frame

Main Objective: Change from baseline of EDS as assessed by mean Maintenance of Wakefulness Test (MWT);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);Primary end point(s): Improvement of Excessive Daytime Sleepiness;Timepoint(s) of evaluation of this end point: after 6 months treatment/intervention

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Secondary

Measure	Time frame
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	—

Measure

Time frame

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

—

Countries

Italy

Contacts

Public ContactAmministrazione Sezione Progetti

Fondazione Serena ONLUS

silvia.pugliatti@centrocliniconemo.it0291433753

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026