* URSA

• Research type

  Research Study

• Full title

  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)

• IRAS ID

  305766

• Contact name

  Sivakumar Sathasivam

• Contact email

  ssathasivam@nhs.net

• Eudract number

  2021-003898-59

• Clinicaltrials.gov Identifier

  NCT05132569

• Clinicaltrials.gov Identifier

  U1111-1265-6378, WHO

• Duration of Study in the UK

  5 years, 0 months, 17 days

• Research summary

  There is an unmet need for people with generalized myasthenia gravis (gMG) for effective treatments with better long-term safety profiles and with more feasible modes of administration.
  Tolebrutinib (SAR442168), a covalent, irreversible inhibitor of Bruton’s tyrosine kinase (BTK), is an immunomodulatory agent relevant to the pathophysiology of gMG that has the advantage of oral administration.

  The purpose of the study is to see if the drug tolebrutinib (SAR442168) works to improve the symptoms of adults with generalized myasthenia gravis (gMG) compared to a group receiving placebo, both groups continuing to receive also their current treatment for gMG. Additional purposes of the study are to find out how safe and tolerable the drug is.

  This is a double blind, placebo controlled study design. Participants will be randomised on a 1:1 ratio to receive the 60mg oral dose of SAR442168 daily or placebo.

  If participants consent, they will attend 15 visit over a 138 week period. The Study comprises of 2 parts.
  A double-blind treatment period of 26 weeks followed by an open-label extension treatment period (for participants having completed the double-blind period), that will provide an additional 2 years of treatment.

  The patient will have blood and urine samples taken, an ECG, physical exam and vital signs will be performed along with completing questionnaires for endpoints.

  The study will include approximately 154 participants, around 4 will be from the UK.
  A pharmaceutical company (Sanofi/Genzyme Corp) is sponsoring this study.

  This study is planned to take part in 2 NHS centres in the UK.

• REC name

  North West - Liverpool Central Research Ethics Committee

• REC reference

  22/NW/0266

• Date of REC Opinion

  2 Nov 2021

• REC opinion

  Further Information Favourable Opinion