Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03914638
Recruitment Status : Recruiting
First Posted : April 15, 2019
Last Update Posted : April 17, 2019
Sponsor:
Information provided by (Responsible Party):
Jan Lykke Scheel Thomsen, University of Aarhus

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.

Condition or disease Intervention/treatment Phase
Myasthenia Gravis Drug: Salbutamol 4Mg Tablet Drug: Placebo oral capsule Phase 2 Phase 3

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).


Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis
Estimated Study Start Date : April 2019
Estimated Primary Completion Date : April 2020
Estimated Study Completion Date : April 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Albuterol

Arm Intervention/treatment
Experimental: Active
Active intervention arm. Treatment for 8 weeks per treatment period.
Drug: Salbutamol 4Mg Tablet
Salbutamol 4 mg, three times daily

Placebo Comparator: Placebo
Placebo arm. Treatment for 8 weeks per treatment period.
Drug: Placebo oral capsule
Placebo, three times daily




Primary Outcome Measures :
  1. Myasthenia Gravis Quality of Life 15-items (MG-QOL15) [ Time Frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2) ]
    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).

  2. Treatment Tolerability [ Time Frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2) ]
    Tolerability assessed by rate of adverse events and drug discontinuation in both treatment periods.


Secondary Outcome Measures :
  1. Myasthenia Gravis Activity of Daily Living (MG-ADL) [ Time Frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2) ]
    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).

  2. Neuro QOL [ Time Frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2) ]
    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).

  3. Quantitative Myasthenia Gravis (QMG) [ Time Frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2) ]
    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).

  4. Myasthenia Gravis Composite (MG-Composite) [ Time Frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2) ]
    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).

  5. Myasthenia Gravis Impairment Index [ Time Frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2) ]
    22 patient-reported and 6 examination items. 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).



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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): NCT03914638


Contacts
Layout table for location contacts
Contact: Jan LS Thomsen, MD 78450000 ext 0045 jathms@rm.dk

Locations
Layout table for location information
Denmark
Department of Neurology, Aalborg University Hospital Not yet recruiting
Aalborg, Denmark, 9000
Neurology, Aarhus University Hospital Recruiting
Aarhus, Denmark, 8200
Sponsors and Collaborators
University of Aarhus
Investigators
Layout table for investigator information
Study Director: Jan LS Thomsen, MD Department of Clinical Medicine, Aarhus University

Layout table for additonal information
Responsible Party: Jan Lykke Scheel Thomsen, MD, PhD Fellow, University of Aarhus
ClinicalTrials.gov Identifier: NCT03914638     History of Changes
Other Study ID Numbers: BETA-MG-01
First Posted: April 15, 2019    Key Record Dates
Last Update Posted: April 17, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
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
Albuterol
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Tocolytic Agents
Reproductive Control Agents
Adrenergic beta-2 Receptor Agonists
Adrenergic beta-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action