Effect of Pyridostigmine (Mestinon) on Muscle Strength in Myasthenia Gravis

NCT03510546 | Unknown Phase 4

• 1 other identifier: Mestinon2018
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 1

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)

Conditions

Myasthenia Gravis

Outcome Measures

Primary Outcomes (1)

• Change in QMG

  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.

  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 (3)

• 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).

• 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).

• 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).

Study Arms (2)

Active

EXPERIMENTAL

De-novo: Each capsule contains 60 mg. pyridostigmine. 1 capsule is administered twice within 4 hours. Chronic: Each capsule contains 60 mg. pyridostigmine. Number of administered capsules per dosage depend on the patient's usual dosage. Study drug is administered twice within 4 hours. Patients are examined/rated before 1st dose, 1 hour after 1st dose, 1 hour after 2nd dose (Visit 1). After cross-over (Visit 2), patients will be rated open-label at 1 month (Visit 3) and 3 months (Visit 4).

Drug: Pyridostigmine

Placebo

PLACEBO COMPARATOR

Same as "Active", however capsules contain placebo.

Drug: Placebo oral capsule

Interventions

Pyridostigmine DRUG

Study dose as per protocol.

Active

Placebo oral capsule DRUG

Study dose as per protocol.

Placebo

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• MG verified by a) anti-body, or b) single-fiber EMG and/or decrement on ENG.

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Aarhus: lead
• Aarhus University Hospital: collaborator

Study Sites (1)

Department of Neurology Aarhus University Hospital

Aarhus, Central Jutland, 8000, Denmark

RECRUITING

Related Publications (2)

• 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.

  PMID: 22622164 BACKGROUND

• 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.

  PMID: 20439845 BACKGROUND

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

Pyridostigmine Bromide

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Pyridinium Compounds; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Jan LS Thomsen, MD

  University of Aarhus

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jan LS Thomsen, MD

CONTACT

+45 7846 6647; jathms@rm.dk

Henning Andersen, MD PhD DMSc

CONTACT

+45 7846 6647; hennande@rm.dk

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD Fellow

Study Record Dates

• First Submitted: April 6, 2018
• First Posted: April 27, 2018
• Study Start: April 9, 2018
• Primary Completion: August 31, 2020
• Study Completion: August 31, 2021
• Last Updated: April 27, 2018

Record last verified: 2018-04

Data Sharing

• IPD Sharing: Will not share

Locations

DK (1)