A Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Rilzabrutinib dose 1, Rilzabrutinib dose 2.
- Who it may be relevant to
- Registry conditions: Graves' Disease. Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Canada, Germany, Italy, Spain, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Open-label Phase 2 Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' Disease
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.
Interventions
- Drug Rilzabrutinib dose 1
Pharmaceutical form:Tablet-Route of administration:Oral - Drug Rilzabrutinib dose 2
Pharmaceutical form:Tablet-Route of administration:Oral
Primary outcome measures
- Percent change from baseline in FT4 levels [Time frame: At Week16]
Secondary outcome measures (4)
- Percent change from baseline in FT3 levels [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]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Italy · 5 centers
- Investigational Site Number : 3800005 — Merano
- Investigational Site Number : 3800001 — Milan
- Investigational Site Number : 3800004 — Milan
- Investigational Site Number : 3800003 — Palermo
- Investigational Site Number : 3800002 — Pisa
Canada · 3 centers
- Investigational Site Number : 1240003 — Calgary
- Investigational Site Number : 1240002 — Surrey
- Investigational Site Number : 1240001 — Sherbrooke
Spain · 3 centers
- Investigational Site Number : 7240002 — Barcelona
- Investigational Site Number : 7240001 — Pamplona
- Investigational Site Number : 7240004 — Seville
Germany · 2 centers
- Investigational Site Number : 2760002 — Essen
- Investigational Site Number : 2760001 — Frankfurt
United Kingdom · 2 centers
- Investigational Site Number : 8260003 — Milton Keynes
- Investigational Site Number : 8260001 — Newcastle upon Tyne
Identifiers
NCT: NCT06984627 · PDY18663 · 2025-521023-75 · U1111-1316-0208