Myasthenia Gravis
Conditions
Brief summary
A randomized, placebo-controlled, double-blinded cross-over study evaluating and quantifying the effect of pyridostigmine on muscle strength and symptoms in Myasthenia Gravis (MG)
Detailed description
The study aims to evaluate and quantify the effect of pyridostigmine on symptoms and muscle strength in newly-diagnosed patients and patients on stable medication. Pyridostigmine treatment is initiated in the vast majority of MG patients. No studies have quantified the effect in a randomized trial, and no studies have examined the potential difference in effect in newly diagnosed patients as compared to patients on stable, antimyasthenic medications. The study will investigate the effect in two groups 1. Newly diagnosed, treatment-naive patients. 2. MG patients on stable antimyasthenic medication.
Interventions
DRUGPyridostigmine
Study dose as per protocol.
DRUGPlacebo oral capsule
Study dose as per protocol.
Sponsors
Aarhus University Hospital
University of Aarhus
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
* MG verified by a) anti-body, or b) single-fiber EMG and/or decrement on ENG.
Exclusion criteria
* Anti-MuSK * Known cardio-pulmonary disease * Known neuropathy * Known myopathy * Known malignant disease * Pregnancy or breastfeeding * Mechanic ileus, urinary tract obstruction, peritonitis De-novo MG Eligibility Criteria * MG diagnosis \< 2 months, no prior antimyasthenic medications Chronic MG Eligibility Criteria * MG diagnosis \> 1 year, and stable pyridostigmine dosis
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in QMG | At baseline, 1 hour after 1st dose (administered immediately after baseline assessment), 1 hour after 2nd dose (administered 2 hours after 1st dose). Assessed on Day 1 and Day 2 (cross-over), at Follow-up 1 (1 month) and Follow-up 2 (3 months). | Quantitative Myasthenia Gravis (QMG) scale rate disease severity of myasthenia gravis on 13 items with a total score ranging from 0-39 (higher values representing more severe disease). Total score (0-39), ocular subitems (0-6), bulbar subitems (0-9), extremity/axial subitems (0-21) and respiratory subitem (0-3) will be reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in muscle strength as assessed by dynamometry (Biodex System 3). | At baseline, 1 hour after 1st dose (administered immediately after baseline assessment), 1 hour after 2nd dose (administered 2 hours after 1st dose). Assessed on Day 1 and Day 2 (cross-over), at Follow-up 1 (1 month) and Follow-up 2 (3 months). | Peak muscle torque expressed in newton meters based on 3 repeated maximal isometric contractions. Performed on shoulder abduction and knee extension. |
| Change in muscle fatigue as assessed by dynamometry (Biodex System 3). | At baseline, 1 hour after 1st dose (administered immediately after baseline assessment), 1 hour after 2nd dose (administered 2 hours after 1st dose). Assessed on Day 1 and Day 2 (cross-over), at Follow-up 1 (1 month) and Follow-up 2 (3 months). | Decrement in peak muscle torque expressed in newton meters per contraction during up to 50 repetitive maximal isokinetic contractions. Performed on shoulder abduction and knee extension. |
| Change in MG Composite Score | At baseline, 1 hour after 1st dose (administered immediately after baseline assessment), 1 hour after 2nd dose (administered 2 hours after 1st dose). Assessed on Day 1 and Day 2 (cross-over), at Follow-up 1 (1 month) and Follow-up 2 (3 months). | The Myasthenia Gravis Composite (MG composite) scale rate disease severity of myasthenia gravis on 10 items with a score ranging from 0-50 (higher values representing more severe disease). |
Countries
Denmark
Contacts
Primary ContactJan LS Thomsen, MD
Backup ContactHenning Andersen, MD PhD DMSc
Outcome results
None listed