Myasthenia GravisPhase 2/3BETA-MGNCT03914638
Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis
A Phase 2/3 study testing Salbutamol 4Mg Tablet in people with Myasthenia Gravis, run by University of Aarhus at 2 sites in Denmark.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis
- What is being tested?
- Salbutamol 4Mg Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 2 sitesDenmark
- Study dates
- 1 Apr 2019to 31 Oct 2021
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
2 sites in 1 country.
Denmark2 sites, 2 recruiting
- Recruiting
Department of Neurology, Aalborg University Hospital
Aalborg, 9000
Izabella Obál, PhD
- Recruiting
Neurology, Aarhus University Hospital
Aarhus, 8200
Jan Thomsen
About this study
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.
Read the 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).
- Conditions
- Myasthenia Gravis
- Being tested
- Salbutamol 4Mg Tablet, Placebo oral capsule
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Salbutamol 4Mg TabletDrug
Salbutamol 4 mg, three times daily
Placebo oral capsuleDrug
Placebo, three times daily
| Study group | Role | What they receive |
|---|---|---|
| Active | Experimental | Active intervention arm. Treatment for 8 weeks per treatment period. Receives: Drug: Salbutamol 4Mg Tablet |
| Placebo | Placebo comparator | Placebo arm. Treatment for 8 weeks per treatment period. Receives: Drug: Placebo oral capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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.
Measured: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Secondary outcomes (4)
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).
Measured: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
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).
Measured: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
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).
Measured: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
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).
Measured: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2019
- Study start1 Apr 2019
- Primary completion31 Oct 2021 (estimated)
- Study completion31 Oct 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia GravisStudies of the same condition, open and under way.
- More trials run by University of AarhusThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Aarhus
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jan Lykke Scheel Thomsen, University of Aarhus
- Investigators
- Jan LS Thomsen, MD, Study director, University of Aarhus
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03914638
- Sponsor's protocol ID
- BETA-MG-01
- Acronym
- BETA-MG
- Status last verified
- Apr 2020
- First submitted
- 11 Apr 2019
- Last updated
- 6 Apr 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT03914638
- Acronym / sponsor ID
- BETA-MG / BETA-MG-01
- Conditions
- Myasthenia Gravis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University of AarhusUniversity, hospital or other
- Study start
- 1 Apr 2019 (actual)
- Primary completion
- 31 Oct 2021 (estimated)
- Study completion
- 31 Oct 2021 (estimated)
- First posted
- 16 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active | Experimental | Drug: Salbutamol 4Mg TabletActive intervention arm. Treatment for 8 weeks per treatment period. |
| Placebo | Placebo comparator | Drug: Placebo oral capsulePlacebo arm. Treatment for 8 weeks per treatment period. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Salbutamol 4Mg TabletSalbutamol 4 mg, three times daily | Drug | Active |
| Placebo oral capsulePlacebo, three times daily | Drug | Placebo |
Primary outcome
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).
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.
4 secondary outcomes
Myasthenia Gravis Activity of Daily Living (MG-ADL)
Time frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Neuro QOL
Time frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Quantitative Myasthenia Gravis (QMG)
Time frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Myasthenia Gravis Composite (MG-Composite)
Time frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jan LS Thomsen, MD study director
University of Aarhus
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Salbutamol 4Mg Tablet in people with Myasthenia Gravis, run by University of Aarhus at 2 sites in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 6 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jan LS Thomsen, MD (78450000 ext. 0045, jathms@rm.dk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Myasthenia Gravis Quality of Life 15-items (MG-QOL15) (time frame: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2020.