search
Back to results

Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis (BETA-MG)

Primary Purpose

Myasthenia Gravis

Status
Unknown status
Phase
Phase 2
Locations
Denmark
Study Type
Interventional
Intervention
Salbutamol 4Mg Tablet
Placebo oral capsule
Sponsored by
University of Aarhus
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myasthenia Gravis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Generalized myasthenia gravis (MGFA IIa-IVb) at screening, verified by ≥ 1 of the following: 1) AchR-antibodies in medical history, 2) Abnormal decrement on repetitive nerve stimulation in medical history
  • Disease duration of ≥ 1 year
  • Stable dose of antimyasthenic medications at screening
  • Residual symptoms with a MG-QOL15 score of ≥ 10
  • Age ≥ 18 years
  • Ability to understand the requirements of the trial and provide written, informed consent

Exclusion Criteria:

  • Evidence of malignancy ≤ 3 years prior to screening, unless deemed completely cured
  • Thymectomy ≤ 6 months prior to screening
  • Impending MG crisis or respiratory insufficiency
  • Worsening of MG symptoms due to other diseases or medications (e.g. infection, beta-blockers, aminoglycosides, etc.)
  • Other factor(s) or medical condition(s) that may explain residual symptoms
  • Pregnancy or breast-feeding
  • Treatment with beta-agonists
  • Uncontrolled diabetes
  • Ischemic Heart Disease, Cardiac Arrhythmia or Heart Failure (including hypertrophic cardiomyopathy)
  • Uncontrolled Hypertension (≥ 160/110)
  • Known hypersensitivity to any of the study drug components
  • Treatment with tricyclic antidepressants, monoamineoxidase inhibitors, digoxine, or methylxanthines.

Sites / Locations

  • Department of Neurology, Aalborg University HospitalRecruiting
  • Neurology, Aarhus University HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Active

Placebo

Arm Description

Active intervention arm. Treatment for 8 weeks per treatment period.

Placebo arm. Treatment for 8 weeks per treatment period.

Outcomes

Primary Outcome Measures

Myasthenia Gravis Quality of Life 15-items (MG-QOL15)
Validated patient reported outcome-questionnaire consisting of 15 items and their impact on quality of life. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Treatment Tolerability
Tolerability assessed by rate of adverse events and drug discontinuation in both treatment periods.

Secondary Outcome Measures

Myasthenia Gravis Activity of Daily Living (MG-ADL)
Validated patient-reported outcome scale consisting of 8 disease-related items and their impact on activity of daily living. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Neuro QOL
Patient reported fatigue-questionnaire used to rate fatigue and impact on quality of life. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Quantitative Myasthenia Gravis (QMG)
Validated rating scale consisting of 13 items measuring muscle function and endurance. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Myasthenia Gravis Composite (MG-Composite)
Validated scale consisting of 10 items with different weighting assessing severity of symptoms in MG. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).

Full Information

First Posted
April 11, 2019
Last Updated
April 2, 2020
Sponsor
University of Aarhus
search

1. Study Identification

Unique Protocol Identification Number
NCT03914638
Brief Title
Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis
Acronym
BETA-MG
Official Title
Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis
Study Type
Interventional

2. Study Status

Record Verification Date
April 2020
Overall Recruitment Status
Unknown status
Study Start Date
April 1, 2019 (Actual)
Primary Completion Date
October 31, 2021 (Anticipated)
Study Completion Date
October 31, 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Aarhus

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This study examines the effect of adjuvant therapy with the oral beta-agonist Salbutamol in patients with generalized myasthenia gravis on stable standard of care having residual symptoms.
Detailed Description
Myasthenia Gravis (MG) causes various degrees of increased muscular fatigue and ocular, bulbar, respiratory and extremity symptoms. Residual symptoms often remain despite treatment with acetylcholinesterase inhibitors and immunosuppressive agents. Escalation of immunosuppressive treatment may provide additional benefit but is associated with potentially severe side effects, and high economic costs. Treatment with beta-agonists has been investigated in animal models of MG, and in small, randomized pilot studies of generalized MG. Adjuvant therapy with oral beta-agonists in MG may be safe and cheap and may improve symptoms. The trial will examine the tolerability and efficacy of adjuvant therapy with the oral beta-agonist Salbutamol in patients with generalized myasthenia gravis on stable standard of care having residual symptoms. Present study is an investigator-initiated, randomized, placebo-controlled, rater and subject-blinded crossover study. Study consists of Screening Period (4 weeks), Treatment Period 1 (8 weeks), Washout Period (4 weeks), Treatment Period 2 (8 weeks).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myasthenia Gravis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Crossover Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Active
Arm Type
Experimental
Arm Description
Active intervention arm. Treatment for 8 weeks per treatment period.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo arm. Treatment for 8 weeks per treatment period.
Intervention Type
Drug
Intervention Name(s)
Salbutamol 4Mg Tablet
Intervention Description
Salbutamol 4 mg, three times daily
Intervention Type
Drug
Intervention Name(s)
Placebo oral capsule
Intervention Description
Placebo, three times daily
Primary Outcome Measure Information:
Title
Myasthenia Gravis Quality of Life 15-items (MG-QOL15)
Description
Validated patient reported outcome-questionnaire consisting of 15 items and their impact on quality of life. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Time Frame
8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Title
Treatment Tolerability
Description
Tolerability assessed by rate of adverse events and drug discontinuation in both treatment periods.
Time Frame
8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Secondary Outcome Measure Information:
Title
Myasthenia Gravis Activity of Daily Living (MG-ADL)
Description
Validated patient-reported outcome scale consisting of 8 disease-related items and their impact on activity of daily living. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Time Frame
8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Title
Neuro QOL
Description
Patient reported fatigue-questionnaire used to rate fatigue and impact on quality of life. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Time Frame
8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Title
Quantitative Myasthenia Gravis (QMG)
Description
Validated rating scale consisting of 13 items measuring muscle function and endurance. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Time Frame
8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Title
Myasthenia Gravis Composite (MG-Composite)
Description
Validated scale consisting of 10 items with different weighting assessing severity of symptoms in MG. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Time Frame
8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Generalized myasthenia gravis (MGFA IIa-IVb) at screening, verified by ≥ 1 of the following: 1) AchR-antibodies in medical history, 2) Abnormal decrement on repetitive nerve stimulation in medical history Disease duration of ≥ 1 year Stable dose of antimyasthenic medications at screening Residual symptoms with a MG-QOL15 score of ≥ 10 Age ≥ 18 years Ability to understand the requirements of the trial and provide written, informed consent Exclusion Criteria: Evidence of malignancy ≤ 3 years prior to screening, unless deemed completely cured Thymectomy ≤ 6 months prior to screening Impending MG crisis or respiratory insufficiency Worsening of MG symptoms due to other diseases or medications (e.g. infection, beta-blockers, aminoglycosides, etc.) Other factor(s) or medical condition(s) that may explain residual symptoms Pregnancy or breast-feeding Treatment with beta-agonists Uncontrolled diabetes Ischemic Heart Disease, Cardiac Arrhythmia or Heart Failure (including hypertrophic cardiomyopathy) Uncontrolled Hypertension (≥ 160/110) Known hypersensitivity to any of the study drug components Treatment with tricyclic antidepressants, monoamineoxidase inhibitors, digoxine, or methylxanthines.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jan LS Thomsen, MD
Phone
78450000
Ext
0045
Email
jathms@rm.dk
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jan LS Thomsen, MD
Organizational Affiliation
Department of Clinical Medicine, Aarhus University
Official's Role
Study Director
Facility Information:
Facility Name
Department of Neurology, Aalborg University Hospital
City
Aalborg
ZIP/Postal Code
9000
Country
Denmark
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Izabella Obál, PhD
Facility Name
Neurology, Aarhus University Hospital
City
Aarhus
ZIP/Postal Code
8200
Country
Denmark
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jan Thomsen

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis

We'll reach out to this number within 24 hrs