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

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ClinicalTrials.gov Identifier: NCT03510546
Recruitment Status : Recruiting
First Posted : April 27, 2018
Last Update Posted : April 27, 2018
Sponsor:
Collaborator:
Aarhus University Hospital
Information provided by (Responsible Party):
Jan Lykke Scheel Thomsen, University of Aarhus

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)

Condition or disease Intervention/treatment Phase
Myasthenia Gravis Drug: Pyridostigmine Drug: Placebo oral capsule Phase 4

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 44 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Effect of Pyridostigmine (Mestinon) on Muscle Strength in Myasthenia Gravis
Actual Study Start Date : April 9, 2018
Estimated Primary Completion Date : August 31, 2020
Estimated Study Completion Date : August 31, 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Active

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
Study dose as per protocol.

Placebo Comparator: Placebo
Same as "Active", however capsules contain placebo.
Drug: Placebo oral capsule
Study dose as per protocol.




Primary Outcome Measures :
  1. Change in QMG [ Time Frame: 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 Outcome Measures :
  1. Change in muscle strength as assessed by dynamometry (Biodex System 3). [ Time Frame: 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.

  2. Change in muscle fatigue as assessed by dynamometry (Biodex System 3). [ Time Frame: 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.

  3. Change in MG Composite Score [ Time Frame: 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).



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Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03510546


Contacts
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Contact: Jan LS Thomsen, MD +45 7846 6647 jathms@rm.dk
Contact: Henning Andersen, MD PhD DMSc +45 7846 6647 hennande@rm.dk

Locations
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Denmark
Department of Neurology Aarhus University Hospital Recruiting
Aarhus, Midtjylland, Denmark, 8000
Contact: Jan LS Thomsen, MD    +45 7846 6647    jathms@rm.dk   
Contact: Henning Andersen, MD PhD DMSc    +45 7846 6647    hennande@rm.dk   
Sponsors and Collaborators
University of Aarhus
Aarhus University Hospital
Investigators
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Principal Investigator: Jan LS Thomsen, MD Department of Clinical Medicine, Aarhus University

Publications:
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Responsible Party: Jan Lykke Scheel Thomsen, MD, PhD Fellow, University of Aarhus
ClinicalTrials.gov Identifier: NCT03510546     History of Changes
Other Study ID Numbers: Mestinon2018
First Posted: April 27, 2018    Key Record Dates
Last Update Posted: April 27, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Pyridostigmine Bromide
Cholinesterase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Cholinergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs