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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

Tracking Information
First Submitted Date  ICMJE April 6, 2018
First Posted Date  ICMJE April 27, 2018
Last Update Posted Date April 27, 2018
Actual Study Start Date  ICMJE April 9, 2018
Estimated Primary Completion Date August 31, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 17, 2018)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: April 17, 2018)
  • 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.
  • 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.
  • 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).
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effect of Pyridostigmine (Mestinon) on Muscle Strength in Myasthenia Gravis
Official Title  ICMJE Effect of Pyridostigmine (Mestinon) on Muscle Strength in Myasthenia Gravis
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Myasthenia Gravis
Intervention  ICMJE
  • Drug: Pyridostigmine
    Study dose as per protocol.
  • Drug: Placebo oral capsule
    Study dose as per protocol.
Study Arms  ICMJE
  • 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).

    Intervention: Drug: Pyridostigmine
  • Placebo Comparator: Placebo
    Same as "Active", however capsules contain placebo.
    Intervention: Drug: Placebo oral capsule
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: April 17, 2018)
44
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 31, 2021
Estimated Primary Completion Date August 31, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 90 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Jan LS Thomsen, MD +45 7846 6647 jathms@rm.dk
Contact: Henning Andersen, MD PhD DMSc +45 7846 6647 hennande@rm.dk
Listed Location Countries  ICMJE Denmark
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03510546
Other Study ID Numbers  ICMJE Mestinon2018
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Jan Lykke Scheel Thomsen, University of Aarhus
Study Sponsor  ICMJE University of Aarhus
Collaborators  ICMJE Aarhus University Hospital
Investigators  ICMJE
Principal Investigator: Jan LS Thomsen, MD Department of Clinical Medicine, Aarhus University
PRS Account University of Aarhus
Verification Date April 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP