Myotonic dystrophy type 1, Steinert disease Myotonic dystrophy type 1
Conditions
Interventions
Mexiletine
Sponsors
Radboud Universitair Medisch Centrum
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: -Clinical and genetic diagnosis of myotonic dystrophy type 1-Age 18-70 years old-Able to walk 10 meters without the support of another person-At least some degree of hand grip myotonia, defined as relaxation time for hand opening after squeezing the fist tightly of = 2 seconds
Exclusion criteria
Exclusion criteria: -Use of pharmaceutical agents that influence muscle relaxation in the last two weeks before the study-Use of pharmaceutical agents that influence the excitability of the cerebral cortex in the last two weeks before the study -Conditions other than DM1 that influence hand function, i.e. Dupuytren -Contraindications for mexiletine use, i.e. untreated second or third-degree heart block, hypersensitivity to the drug or drug class-Contraindication for transcranial magnetic stimulation as defined in the Standard Operating Procedure (SOP) for Non-Invasive Brain Stimulation (2021) of the Donders Institute for Brain, Cognition and Behaviour -Use of an experimental drug in a DM1 clinical trial within the past 90 days-Pregnancy or active wish to conceive.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Handgrip relaxation time (via transcranial magnetic stimulation, muscle ultrasound, video hand opening time, time to go from 90% to 5% of peak handgrip force, vHOT). | — |
Secondary
| Measure | Time frame |
|---|---|
| Muscular impairment rating scale (MIRS), handgrip strength, functional tests (9-hole peg test (HPT), 10-meter walk/run test (MWT), timed test of water swallowing (TWST), timed test of mastication (TOMASS)), patient-reported measures (DM1-Activ, MD-Health Index, Patient Global Impression of Change (PGIC)). | — |
Countries
Netherlands
Contacts
Public ContactK. Mul
Radboud Universitair Medisch Centrum
Outcome results
None listed