Efficacy and Safety of Tolebrutinib (SAR442168) Tablets in Adult Participants With Generalized Myasthenia Gravis

Titre officiel

A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Tolebrutinib (SAR442168) in Adults With Generalized Myasthenia Gravis (MG)

Sommaire:

This is a multicentre, randomized, double blind, placebo-controlled, Phase 3 study to evaluate the efficacy and safety of tolebrutinib compared with placebo in adult participants aged 18 to 85 years old with moderate-to-severe gMG, who are receiving Standard of Care (SoC). The double-blind (DB) treatment period of 26 weeks will comprise of 7 site visits followed by a 2-year open label extension (OLE) period with quarterly visits. The efficacy of tolebrutinib versus placebo during DB period will be assessed by clinical evaluations, that include scales based on physician examination or direct participant feedback i.e., patient reported outcomes (PROs). These evaluations will continue during the OLE in order to measure long term efficacy and safety.

Description de l'essai

Primary Outcome:

• DB period: Change from baseline in Myasthenia gravis-activities of daily living (MG-ADL) total score
• OLE: Number of participants with adverse events (AEs) /SAEs

Secondary Outcome:

• Change from baseline in Quantitative MG (QMG) total score
• Change from baseline in MG Impairment Index (MGII) total score
• Change from baseline in MG Quality of Life Questionnaire (MG-QOL15) survey score
• Proportion of participants with ≥ 2-point reduction from baseline in MG-ADL total score
• Proportion of participants with ≥ 3-point reduction from baseline in QMG total score
• DB: Number of participants with AEs /SAEs
• OLE: Change from baseline in MG-ADL total score
• OLE: Proportion of participants achieving a reduction from baseline in daily dose of oral corticosteroids (OCS)

The duration of the DB period will be 26 weeks. The OLE is up to 104 weeks. The duration of the whole study DB+OLE is 130 weeks.

Voir cet essai sur ClinicalTrials.gov