Efficacy and Safety of Tolebrutinib (SAR442168) Tablets in Adult Participants With Generalized Myasthenia Gravis (URSA)
Brief description of study
This is 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 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.
Detailed description of study
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.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Myasthenia Gravis
Age: Between 18 Years - 85 Years
Gender: Male or Female
- Participants must be 18 years of age to 85 years of age inclusive, at the time of signing the informed consent
- Participants with a diagnosis of gMG at screening with generalized muscle weakness meeting the clinical criteria for diagnosis of MG, as defined by the MGFA Clinical Classification Class II, III, or IV, and likely not in need of a respirator for the duration of the study, as judged by the Investigator
- Positive serologic testing for anti-AChR or anti-MuSK autoantibody at screening OR
- Seronegative for both anti-AChR and anti-MuSK autoantibodies and with prior diagnosis
supported by ≥1 of the following 3 tests:
- History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation
- History of positive edrophonium chloride test
- Participant has demonstrated improvement in gMG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician.
- The participant must have a total score ≥6 on MG-ADL scale at screening and D1 with
greater than half of the score attributed to non-ocular items
- MGFA Class I (ocular MG) or Class V
- Participants having undergone thymectomy within 6 months of screening or having a planned thymectomy during the trial period.
- The participant has a history of infection or may be at risk for infection: A history of active or latent tuberculosis (TB); Participants at risk of developing or having reactivation of hepatitis; Persistent chronic or active recurring infection requiring treatment with antibiotics, antivirals, or antifungals; Fever within 4 weeks of the Screening Visit (≥38°C; however, if due to brief and mild ear, nose, throat viral infection participant may be included based on the Investigator's judgment); A history of infection with human immunodeficiency virus (HIV); A history of T-lymphocyte or T-lymphocyte-receptor vaccination, transplantation (including solid organ, stem cell, and bone marrow transplantation) and/or antirejection therapy
- Any malignancy within the past 5 years prior Screening Visit (except for effectively treated carcinoma in situ of the cervix, adequately treated non-metastatic squamous or basal cell carcinoma of the skin and malignant thymoma that have been resected or are considered as cured by any treatment with no evidence of metastatic disease for ≥3 years) will be exclusionary
- Conditions that may predispose the participant to excessive bleeding
- Clinically significant laboratory abnormalities (including evidence of liver injury) or electrocardiogram abnormalities at Screening
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
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