Clinical trial NCT06984627: A Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' Disease
Official title: An Open-label Phase 2 Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' Disease
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Graves' Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2025-05-22
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Percent change from baseline in FT4 levels
timeFrame: At Week16
Secondary outcomes:
measure: Percent change from baseline in FT3 levels
timeFrame: At Week16
measure: Proportion of participants with FT4 levels within normal limits
timeFrame: At Week16
measure: Proportion of participants with FT3 levels within normal limits
timeFrame: At Week16
measure: 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
timeFrame: Up to week 20
Other outcomes:
Locations:
Study officials:
References: