RILZABRUTINIB for Graves' disease|Graves' orbitopathy

Inclusion criteria

• {"criterion_text":"- Participants who have a confirmed diagnosis of Graves’ disease with active hyperthyroidism, with or without active Graves’ orbitopathy at the time of screening"}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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

• {"criterion_text":"- History of hyperthyroidism not caused by Graves’ disease (eg, hyperthyroidism due to toxic multinodular goiter, autonomous thyroid nodule, acute inflammatory thyroiditis)."}
• {"criterion_text":"- History of thyroid storm or at high risk of developing thyroid storm as determined by evaluating clinician."}
• {"criterion_text":"- Enlarged thyroid goiter causing upper airway obstruction and/or requiring surgical intervention during the study period."}
• {"criterion_text":"- For participant with Graves’ orbitopathy, requires immediate surgical ophthalmological intervention or is planning corrective surgery/irradiation during the course of the study."}
• {"criterion_text":"- Sight threatening Graves’ orbitopathy or decreased visual acuity due to optic neuropathy within the last 6 months."}
• {"criterion_text":"- Corneal decompensation unresponsive to medical management."}
• {"criterion_text":"- Onset of Graves’ orbitopathy symptoms >9 months prior to baseline."}

Primary endpoints

• {"endpoint_text":"- Percent change from baseline in FT4 levels","definition_or_measurement_approach":"Measured as percent change from baseline in FT4 levels (free thyroxine)."}

Secondary endpoints

• {"endpoint_text":"- Percent change from baseline in FT3 levels","definition_or_measurement_approach":"Measured as percent change from baseline in FT3 levels (free triiodothyronine)."}
• {"endpoint_text":"- Proportion of participants with FT4 levels within normal limits","definition_or_measurement_approach":"Proportion of participants whose FT4 levels fall within laboratory normal limits."}
• {"endpoint_text":"- Proportion of participants with FT3 levels within normal limits","definition_or_measurement_approach":"Proportion of participants whose FT3 levels fall within laboratory normal limits."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Incidence counts/rates of TEAEs, SAEs, AESIs and withdrawals due to TEAEs occurring during the study period as recorded in safety reporting."}

Methods

• Poster — K2-recruitment-material-poster-de / K2-recruitment-material-poster-es / K2-recruitment-material-poster-it (country-specific posters for Germany, Spain, Italy)
• Doctor-to-doctor letter — K2-recruitment-material-dr-to-dr-letter-de / -es / -it (materials aimed at HCP referral in Germany, Spain, Italy)
• Patient brochure — K2-recruitment-material-patient-brochure-de / -es / -it (patient-facing brochures in Germany, Spain, Italy)
• Referral letter for HCP — K2-recruitment-material-referral-letter-hcp-de / -es / -it (referral letters to HCPs in Germany, Spain, Italy)
• Social media post — K2-recruitment-material-social-media-post-it-it (social media recruitment material in Italy)
• Recruitment arrangements document (K1) available in English (K1-recruitment-arrangements-en)

Germany

Earliest CTIS Part Ii Submission Date

07-07-2025

Latest Decision Or Authorization Date

14-08-2025

Processing Time Days

38

Number Of Sites

2

Number Of Participants

8

Spain

Earliest CTIS Part Ii Submission Date

23-06-2025

Latest Decision Or Authorization Date

29-01-2026

Processing Time Days

220

Number Of Sites

3

Number Of Participants

10

Italy

Earliest CTIS Part Ii Submission Date

23-06-2025

Latest Decision Or Authorization Date

23-03-2026

Processing Time Days

273

Number Of Sites

5

Number Of Participants

12

Primary sponsor

Full Name

Sanofi-Aventis Recherche & Developpement

Organisation Type

Pharmaceutical company

Country Of Registered Address

France

Third parties

• {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"long-term storage","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Marken","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Denmark","full_name":"Clinigma ApS","duties_or_roles":"In-trial qualitative interviews","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"long-term storage","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}