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

NCT06984627 | Recruiting Phase 2 FDA Drug

• 3 other identifiers: PDY186632025-521023-75U1111-1316-0208
• Study Type: interventional
• Target: 30
• Locations: 5 countries
• Sites: 13

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.

Conditions

Graves' Disease

Outcome Measures

Primary Outcomes (1)

• Percent change from baseline in FT4 levels

  Free thyroxine

  At Week16

Secondary Outcomes (4)

• Percent change from baseline in FT3 levels

  At Week16

• Proportion of participants with FT4 levels within normal limits

  At Week16

• Proportion of participants with FT3 levels within normal limits

  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

  Up to week 20

Study Arms (2)

Rilzabrutinib dose 1

EXPERIMENTAL

Rilzabrutinib

Drug: Rilzabrutinib dose 1

Rilzabrutinib dose 2

EXPERIMENTAL

Rilzabrutinib

Drug: Rilzabrutinib dose 2

Interventions

Rilzabrutinib dose 2 DRUG

Pharmaceutical form:Tablet-Route of administration:Oral

Also known as: SAR444671

Rilzabrutinib dose 2

Rilzabrutinib dose 1 DRUG

Pharmaceutical form:Tablet-Route of administration:Oral

Also known as: SAR444671

Rilzabrutinib dose 1

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (13)

Investigational Site Number : 1240003

Calgary, Alberta, T2N 4L7, Canada

RECRUITING

Investigational Site Number : 1240002

Surrey, British Columbia, V3T 4G8, Canada

RECRUITING

Investigational Site Number : 1240001

Sherbrooke, Quebec, J1H 5N4, Canada

RECRUITING

Investigational Site Number : 2760002

Essen, 45147, Germany

RECRUITING

Investigational Site Number : 2760001

Frankfurt, 60590, Germany

RECRUITING

Investigational Site Number : 3800001

Milan, Milano, 20122, Italy

RECRUITING

Investigational Site Number : 3800003

Palermo, 90127, Italy

RECRUITING

Investigational Site Number : 3800002

Pisa, 56126, Italy

RECRUITING

Investigational Site Number : 7240002

Barcelona, Catalunya [Cataluña], 08036, Spain

RECRUITING

Investigational Site Number : 7240001

Pamplona, Navarre, 31008, Spain

RECRUITING

Investigational Site Number : 7240004

Seville, Sevilla, 41009, Spain

RECRUITING

Investigational Site Number : 8260003

Milton Keynes, Buckinghamshire, MK6 5LD, United Kingdom

RECRUITING

Investigational Site Number : 8260001

Newcastle upon Tyne, England, NE2 4HH, United Kingdom

RECRUITING

Related Links

• PDY18663 Plain Language Results Summary

MeSH Terms

Conditions

Graves Disease

Condition Hierarchy (Ancestors)

Exophthalmos; Orbital Diseases; Eye Diseases; Goiter; Thyroid Diseases; Endocrine System Diseases; Hyperthyroidism; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610; Contact-US@sanofi.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 14, 2025
• First Posted: May 22, 2025
• Study Start: September 2, 2025
• Primary Completion (Estimated): August 25, 2026
• Study Completion (Estimated): September 23, 2026
• Last Updated: May 22, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

GB (2); DE (2); IT (3); ES (3); CA (3)