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

Investigational Study on the Efficacy and Safety of Investigational Medication in Adults with Graves' Disease

Recruiting
18 years - 75 years
All
Phase 2
30 participants needed

Study Overview

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.

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Graves' Disease
  • Age: 18 years - 75 years
  • Gender: All

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.

This study investigates the effects of an investigational medication in adults with Graves' disease.

The purpose of this study is to evaluate how well the investigational medication works and its safety in people with Graves' disease. The study will focus on understanding the overall impact of the medication on the condition.

  • Who can participate: Adults aged 18 years or older with a confirmed diagnosis of Graves' disease, with or without active Graves' orbitopathy, can participate. Key eligibility includes active hyperthyroidism and specific criteria for those with Graves' orbitopathy.
  • Study details: Participants will be involved in various activities related to the investigational medication. The focus will be on monitoring the medication's effects and ensuring participant safety throughout the study. A placebo, an inactive substance that looks like the investigational medicine but does not contain any medicine, may be used for comparison.
  • Study timelines and visits: The study will last up to 24 weeks. The study requires up to 13 visits.
Updated on 13 Mar 2026. Study ID: NCT06984627

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