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

Investigating the Effects of an Investigational Medication in Graves' Disease (Overactive Thyroid)

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

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.

Updated on 11 Sep 2025. Study ID: NCT06984627

This study investigates the effects and safety of an investigational medication on individuals with Graves' disease, which is an autoimmune disorder that causes the thyroid gland to produce excessive hormones. Some participants may also have Graves' orbitopathy, a condition where the tissues around the eyes become inflamed. The study involves two different doses of the investigational medication and includes participants aged 18 years or older.

Participants will undergo various procedures, including an ophthalmic exam to assess the severity of Graves' orbitopathy, if present. The study is divided into several periods: a screening period of up to 4 weeks, a treatment period of up to 16 weeks, and a follow-up period of 4 weeks. The total number of visits required during the study is up to 13.

  • Who can participate: Adults aged 18 years or older with a confirmed diagnosis of Graves' disease, with or without active Graves' orbitopathy, can participate. Participants must have active hyperthyroidism and meet specific ophthalmic criteria for Graves' orbitopathy, if applicable.
  • Study details: Participants will receive one of two doses of the investigational medication. The study will include regular assessments and monitoring to evaluate the effects of the treatment. A placebo is not used in this study.
  • Study timelines and visits: The study will last up to 24 weeks. The study requires up to 13 visits.

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