Clinical trial NCT03510546: Effect of Pyridostigmine (Mestinon) on Muscle Strength in Myasthenia Gravis
Official title: Effect of Pyridostigmine (Mestinon) on Muscle Strength in Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Myasthenia Gravis
Lead sponsor: University of Aarhus
Sponsor class: OTHER
Collaborators: name: Aarhus University Hospital
First posted: 2018-04-27
Enrollment: 44
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in QMG
timeFrame: 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).
Secondary outcomes:
measure: Change in muscle strength as assessed by dynamometry (Biodex System 3).
timeFrame: 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).
measure: Change in muscle fatigue as assessed by dynamometry (Biodex System 3).
timeFrame: 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).
measure: Change in MG Composite Score
timeFrame: 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).
Other outcomes:
Locations:
city: Aarhus
country: Denmark
facility: Department of Neurology Aarhus University Hospital
state: Midtjylland
Study officials:
affiliation: University of Aarhus
name: Jan LS Thomsen, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Barnett C, Katzberg H, Nabavi M, Bril V. The quantitative myasthenia gravis score: comparison with clinical, electrophysiological, and laboratory markers. J Clin Neuromuscul Dis. 2012 Jun;13(4):201-5. doi: 10.1097/CND.0b013e31824619d5.
citation: Burns TM, Conaway M, Sanders DB; MG Composite and MG-QOL15 Study Group. The MG Composite: A valid and reliable outcome measure for myasthenia gravis. Neurology. 2010 May 4;74(18):1434-40. doi: 10.1212/WNL.0b013e3181dc1b1e.