NCT05132569

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

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)

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

This was a multicenter, 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 generalized myasthenia gravis (gMG), who received Standard of Care (SoC). The double-blind (DB) treatment period of 26 weeks comprised of 7 site visits followed by a 2-year open label extension (OLE) period with quarterly visits. The efficacy of tolebrutinib versus placebo during the DB period was assessed by clinical evaluations, including scales based on physician examination or direct participant feedback i.e., patient reported outcomes (PROs). These evaluations continued during the OLE to measure long term efficacy and safety.

Detailed description

The duration of the DB period was 26 weeks. The OLE was planned up to 104 weeks. The duration of the whole study DB+OLE was planned up to130 weeks.

Conditions

• Myasthenia Gravis

Interventions

Type	Name	Description
DRUG	Tolebrutininb	Pharmaceutical form: Film-coated tablet Route of administration: Oral
DRUG	Placebo	Pharmaceutical form: Film-coated tablet Route of administration: Oral

Timeline

• Start date: 2021-12-03
• Primary completion: 2023-02-21
• Completion: 2023-02-21
• First posted: 2021-11-24
• Last updated: 2025-09-09
• Results posted: 2024-04-08

Locations

22 sites across 9 countries: United States, Canada, China, Hungary, Italy, Japan, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study