PDY18663
Research type
Research Study
Full title
An open-label Phase 2 study to investigate the efficacy and safety of rilzabrutinib in adult participants with Graves’ disease
IRAS ID
1011908
Contact name
Patrick Maury
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Eudract number
2025-521023-75
Research summary
Graves’ disease is a condition characterized by thyroid gland dysfunction due to the presence of autoantibodies. Autoantibodies are immune system proteins that target the body’s own cells, causing the body to make too much thyroid hormone (hyperthyroidism), which affects many organs in the body. Thyroid eye disease, also known as Graves’ orbitopathy, occurs frequently in people with Graves’ disease. Treatment with rilzabrutinib may decrease the level of autoantibodies, preventing the excessive production of thyroid hormone & reducing disease symptoms. This study is designed to investigate the safety and efficacy of rilzabrutinib in patients with Graves’ disease.
The main goal is to explore the effect of rilzabrutinib treatment in reducing thyroid gland activity. Other goals include exploring the effect of rilzabrutinib treatment in reducing the thyroid gland activity and to assess the side effects.
This study is an open-label, 2-arm study where each person participates for 24 weeks. Open-label means that the participants and researchers both know which treatment is given. The study contains a screening period (up to 4 weeks), an open-label rilzabrutinib treatment period (up to 16 weeks), and a safety follow-up period (4 weeks).
Rilzabrutinib is being studied as a possible treatment for Graves’ disease. It is thought to work by blocking a protein called Bruton’s tyrosine kinase (BTK), thereby preventing activation of immune cells that produce the autoantibodies that cause thyroid gland dysfunction. 2 different doses of rilzabrutinib will be tested. Participants will take one selected dose of the study medicine, by mouth. The inclusion criteria for this study include adults at least 18 years of age with a confirmed diagnosis of Graves’ disease. Some of the participants will also have Graves’ orbitopathy. The exclusion criteria of this study will include people with certain medical conditions, or anything else that makes them unsuitable for the study.REC name
North East - York Research Ethics Committee
REC reference
25/NE/0049
Date of REC Opinion
13 May 2025
REC opinion
Further Information Favourable Opinion