Rilzabrutinib treatment for adults with Graves' disease
An Open-label Phase 2 Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' Disease
This trial will test two doses of the oral drug rilzabrutinib in adults with Graves' disease, with or without Graves' orbitopathy, to see which dose better controls hyperthyroidism and is safe.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | Sanofi Industry-sponsored |
| Drugs / interventions | rilzabrutinib |
| Locations | 13 sites (Calgary, Alberta and 12 other locations) |
| Trial ID | NCT06984627 on ClinicalTrials.gov |
What this trial studies
This is a Phase 2, parallel-group, two-arm study comparing two doses of rilzabrutinib in adults (age ≥18) who have active Graves' disease, with or without active Graves' orbitopathy. Participants undergo up to 4 weeks of screening, up to 16 weeks of treatment, and a 4-week follow-up, with as many as 13 clinic visits. A subset of participants will have active Graves' orbitopathy confirmed by ophthalmic exam and a clinical activity score (CAS) ≥3. The main focus is to measure the treatment effect and safety of each dose over the treatment period.
Who should consider this trial
Good fit: Adults (≥18 years) with a confirmed diagnosis of Graves' disease and active hyperthyroidism, including those with or without active Graves' orbitopathy (a subset requires CAS ≥3).
Not a fit: People whose hyperthyroidism is caused by conditions other than Graves' disease or those at high risk for thyroid storm are not expected to benefit from this study.
Why it matters
Potential benefit: If successful, rilzabrutinib could provide a new oral option that helps control hyperthyroidism and may improve orbitopathy symptoms with an acceptable safety profile.
How similar studies have performed: Using BTK inhibitors like rilzabrutinib for Graves' disease is relatively novel; related BTK inhibitors have shown promise in other autoimmune conditions but are not established treatments for Graves' disease.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participants who have a confirmed diagnosis of Graves' disease with active hyperthyroidism, with or without active Graves' orbitopathy at the time of screening. * A subset of participants will have a diagnosis of active Graves' orbitopathy, as confirmed by ophthalmic exam at screening and a clinical activity score (CAS) ≥3 for the most severely affected eye, and associated with one or more of the following: lid retraction ≥2 mm, moderate or severe soft tissue involvement, proptosis ≥2 mm, and/or intermittent or constant diplopia. * Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Exclusion Criteria: * History of hyperthyroidism not caused by Graves' disease (eg, hyperthyroidism due to toxic multinodular goiter, autonomous thyroid nodule, acute inflammatory thyroiditis). * History of thyroid storm or at high risk of developing thyroid storm as determined by evaluating clinician. * Enlarged thyroid goiter causing upper airway obstruction and/or requiring surgical intervention during the study period. * For participant with Graves' orbitopathy, requires immediate surgical ophthalmological intervention or is planning corrective surgery/irradiation during the course of the study. * Sight threatening Graves' orbitopathy or decreased visual acuity due to optic neuropathy within the last 6 months. * Corneal decompensation unresponsive to medical management. * Onset of Graves' orbitopathy symptoms \>9 months prior to baseline. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Where this trial is running
Calgary, Alberta and 12 other locations
- Investigational Site Number : 1240003 — Calgary, Alberta, Canada (Recruiting)
- Investigational Site Number : 1240002 — Surrey, British Columbia, Canada (Recruiting)
- Investigational Site Number : 1240001 — Sherbrooke, Quebec, Canada (Recruiting)
- Investigational Site Number : 2760002 — Essen, Germany (Recruiting)
- Investigational Site Number : 2760001 — Frankfurt, Germany (Recruiting)
- Investigational Site Number : 3800001 — Milan, Milano, Italy (Recruiting)
- Investigational Site Number : 3800003 — Palermo, Italy (Recruiting)
- Investigational Site Number : 3800002 — Pisa, Italy (Recruiting)
- Investigational Site Number : 7240002 — Barcelona, Catalunya [Cataluña], Spain (Recruiting)
- Investigational Site Number : 7240001 — Pamplona, Navarre, Spain (Recruiting)
- Investigational Site Number : 7240004 — Seville, Sevilla, Spain (Recruiting)
- Investigational Site Number : 8260003 — Milton Keynes, Buckinghamshire, United Kingdom (Recruiting)
- Investigational Site Number : 8260001 — Newcastle upon Tyne, England, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)
- Email: Contact-US@sanofi.com
- Phone: 800-633-1610
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.