A study to investigate the efficacy and safety of rilzabrutinib in adult participants with Graves’ disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Sanofi-Aventis Recherche & Developpement

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

3 Oct 2025 → ongoing

Decision date (initial)

2025-07-16

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2025-521023-75-00

WHO UTN

U1111-1316-0208

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacogenetic, Pharmacokinetic, Efficacy, Therapy, Safety, Pharmacodynamic

To explore the treatment effect of rilzabrutinib in reducing thyroid activity in participants with Graves’ disease

Secondary objectives 2

1. To explore the treatment effect of rilzabrutinib in reducing thyroid activity in participants with Grave’s disease
2. To evaluate the safety of rilzabrutinib.

Conditions and MedDRA coding

Immune system diseases

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Participants who have a confirmed diagnosis of Graves’ disease with active hyperthyroidism, with or without active Graves’ orbitopathy at the time of screening
2. 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.
3. 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 7

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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Percent change from baseline in FT4 levels

Secondary endpoints 4

1. Percent change from baseline in FT3 levels
2. Proportion of participants with FT4 levels within normal limits
3. Proportion of participants with FT3 levels within normal limits
4. 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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Sanofi-Aventis Recherche & Developpement

Sponsor organisation

Sanofi-Aventis Recherche & Developpement

Address

82 Avenue Raspail

City

Gentilly

Postcode

94250

Country

France

Scientific contact point

Organisation

Sanofi-Aventis Recherche & Developpement

Contact name

Clinical Sciences and Operations

Public contact point

Organisation

Sanofi-Aventis Recherche & Developpement

Contact name

Clinical Sciences and Operations

Third parties 6

Locations

3 EU/EEA countries · 10 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 30 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

6 submissions — initial application plus substantial / non-substantial modifications