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

ConditionGraves' Disease

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18-75

SponsorSanofi

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Parallel

Treatments tested in this trial

Rilzabrutinib dose 1
Drug
Pharmaceutical form:Tablet-Route of administration:Oral
Rilzabrutinib dose 2
Drug
Pharmaceutical form:Tablet-Route of administration:Oral

Treatment groups

30 Participants

are divided into 2 treatment groups

Group A: Rilzabrutinib dose 1Experimental treatment 1 intervention

Group B: Rilzabrutinib dose 2Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

Percent change from baseline in FT4 levels

Free thyroxine

Time frame

At Week16

Secondary outcomes

Percent change from baseline in FT3 levels

Free triiodothyronine

Time frame

At Week16

Proportion of participants with FT4 levels within normal limits

Time frame

At Week16

Proportion of participants with FT3 levels within normal limits

Time frame

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

Time frame

Up to week 20