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

Placebo arm. Treatment for 8 weeks per treatment period.

Salbutamol 4Mg Tablet

Placebo oral capsule

Jan LS Thomsen, MD

Izabella Obál, PhD

Jan Thomsen

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.

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).

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

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).

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

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).

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).

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).

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).

University of Aarhus

MD, PhD Fellow

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis	NCT03914638	Myasthenia Grav...	Salbutamol 4Mg ... Placebo oral ca...	18 Years -	University of Aarhus
Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis	NCT03914638	Myasthenia Grav...	Salbutamol 4Mg ... Placebo oral ca...	18 Years -	University of Aarhus

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