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

• Register: EUCTR
• Last refreshed on: 17 August 2021
• Main ID: EUCTR2016-000601-36-IT
• Date of registration: 08/06/2021
• Prospective Registration: No
• Primary sponsor: FONDAZIONE SERENA ONLUS
• Public title: Clinical trial on the efficacy of non invasive ventilation and modafinil on excessive daytime sleepiness in Myotonic Dystrophy type 1
• Scientific title: CLINICAL EFFICACY OF NIV AND MODAFINIL ON EXCESSIVE DAYTIME SLEEPINESS: A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL IN DM1 - ModaNIV
• Date of first enrolment: 26/07/2016
• Target sample size: 80
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-000601-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Italy

Contacts

• Name: Amministrazione Sezione Progetti
• Affiliation: Fondazione Serena ONLUS

• Name: Amministrazione Sezione Progetti
• Affiliation: Fondazione Serena ONLUS

Key inclusion & exclusion 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:
(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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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]

Intervention(s)

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

Primary Outcome(s)

Primary end point(s): Improvement of Excessive Daytime Sleepiness

Timepoint(s) of evaluation of this end point: after 6 months treatment/intervention

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)

Secondary Outcome(s)

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

Secondary ID(s)

GUP15004

Source(s) of Monetary Support

Telethon

Ethics review

Status: Approved
Approval date: 22/04/2016
Contact: