NCT03914638

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Apr 2019

Geographic Reach
1 country

2 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2019

Completed
10 days until next milestone

First Submitted

Initial submission to the registry

April 11, 2019

Completed
5 days until next milestone

First Posted

Study publicly available on registry

April 16, 2019

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2021

Completed
Last Updated

April 6, 2020

Status Verified

April 1, 2020

Enrollment Period

2.6 years

First QC Date

April 11, 2019

Last Update Submit

April 2, 2020

Conditions

Myasthenia Gravis

Outcome Measures

Primary Outcomes (2)

  • 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).

    8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)

  • Treatment Tolerability

    Tolerability assessed by rate of adverse events and drug discontinuation in both treatment periods.

    8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)

Secondary Outcomes (4)

  • Myasthenia Gravis Activity of Daily Living (MG-ADL)

    8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)

  • Neuro QOL

    8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)

  • Quantitative Myasthenia Gravis (QMG)

    8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)

  • Myasthenia Gravis Composite (MG-Composite)

    8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)

Study Arms (2)

Active

EXPERIMENTAL

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

Drug: Salbutamol 4Mg Tablet

Placebo

PLACEBO COMPARATOR

Placebo arm. Treatment for 8 weeks per treatment period.

Drug: Placebo oral capsule

Interventions

Salbutamol 4Mg TabletDRUG

Salbutamol 4 mg, three times daily

Active
Placebo oral capsuleDRUG

Placebo, three times daily

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Department of Neurology, Aalborg University Hospital

Aalborg, 9000, Denmark

RECRUITING

Neurology, Aarhus University Hospital

Aarhus, 8200, Denmark

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

AlbuterolTablets

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous SystemNervous System NeoplasmsNeoplasms by SiteNeoplasmsParaneoplastic SyndromesAutoimmune Diseases of the Nervous SystemNervous System DiseasesNeurodegenerative DiseasesNeuromuscular Junction DiseasesNeuromuscular DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

EthanolaminesAmino AlcoholsAlcoholsOrganic ChemicalsAminesPhenethylaminesEthylaminesDosage FormsPharmaceutical Preparations

Study Officials

  • Jan LS Thomsen, MD

    University of Aarhus

    STUDY DIRECTOR

Central Study Contacts

Jan LS Thomsen, MD

CONTACT

78450000jathms@rm.dk

Study Design

Study Type
interventional
Phase
phase 2
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 11, 2019

First Posted

April 16, 2019

Study Start

April 1, 2019

Primary Completion

October 31, 2021

Study Completion

October 31, 2021

Last Updated

April 6, 2020

Record last verified: 2020-04

Data Sharing

IPD Sharing
Will not share

Locations

DK(2)