A Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' Disease

Phase 2

Not yet recruiting

• Conditions: Graves' Disease
• Interventions: Drug: Rilzabrutinib dose 1; Drug: Rilzabrutinib dose 2

• Registration Number: NCT06984627
• Lead Sponsor: Sanofi

Brief Summary

This is a parallel group, Phase 2, 2-arm study to measure the treatment effect and safety of rilzabrutinib dose 1 or rilzabrutinib dose 2 in participants with Graves' disease, with and without Graves' orbitopathy, aged 18 years or older.

Study details include:

* Screening period (up to 4 weeks).

* Treatment period (up to 16 weeks).

* Follow-up period (4 weeks). The number of visits will be up to 13.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-05
• Study First Submitted: 2025-05-14
• Study First Submitted QC: 2025-05-14
• Last Update Submitted: 2025-05-14
• Study First Posted: 2025-05-22
• Last Update Posted: 2025-05-22
• Study Start: 2025-05-30
• Primary Completion: 2026-08-25
• Study Completion: 2026-09-23

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 30

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Rilzabrutinib dose 1	Rilzabrutinib dose 1	Rilzabrutinib
Rilzabrutinib dose 2	Rilzabrutinib dose 2	Rilzabrutinib

Primary Outcome Measures

Name	Time	Method
Percent change from baseline in FT4 levels	At Week16	Free thyroxine

Secondary Outcome Measures

Name	Time	Method
Percent change from baseline in FT3 levels	At Week16	Free triiodothyronine
Proportion of participants with FT4 levels within normal limits	At Week16
Proportion of participants with FT3 levels within normal limits	At Week16
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs) and withdrawals due to TEAEs during the study period	Up to week 20