Study on the Effects and Safety of Rilzabrutinib for Adults with Graves’ Disease

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What is this study about?

This study evaluates the effectiveness and safety of rilzabrutinib as a treatment for Graves’ disease. Graves’ disease is an autoimmune disorder that causes the thyroid gland to produce too much thyroid hormone (hyperthyroidism). This excess thyroid activity can lead to symptoms such as rapid heartbeat, weight loss, anxiety, and in some cases, eye problems known as Graves’ orbitopathy, which can cause bulging eyes, double vision, and eyelid retraction.

The purpose of this research is to determine if rilzabrutinib can reduce thyroid activity in people with Graves’ disease. The study is open-label, which means both the participants and researchers know which treatment is being given. The medication targets specific components of the immune system that are involved in the disease process.

During the study, participants will receive rilzabrutinib and be monitored for changes in thyroid hormone levels (FT3 and FT4) and any side effects that may occur. Some participants in the study will have the eye-related symptoms of Graves’ disease (Graves’ orbitopathy). The researchers will evaluate how the medication affects both the thyroid function and these eye-related symptoms.

1 Initial Screening

Your participation begins with confirming your Graves’ disease diagnosis with active hyperthyroidism. Some participants may also have active Graves’ orbitopathy, which affects the eyes.

If you have Graves’ orbitopathy, this will be confirmed through an eye examination and a clinical activity score of 3 or higher in your most affected eye, along with symptoms such as lid retraction, soft tissue involvement, proptosis (bulging eyes), or double vision.

2 Starting Treatment

You will receive rilzabrutinib, which comes as a film-coated tablet. This is an open-label study, which means both you and the study doctor will know which medication you are taking.

The study will monitor how rilzabrutinib affects your thyroid activity. You will need to take the medication as prescribed by your study doctor for the duration of the trial.

3 Regular Monitoring

Throughout the study, your thyroid hormone levels will be measured regularly, specifically Free T4 (FT4) and Free T3 (FT3) levels. These are hormones produced by your thyroid gland that are typically elevated in Graves’ disease.

The study will track the percent change in these hormone levels from your starting values, and whether they return to normal ranges during treatment.

4 Safety Assessments

You will undergo regular safety assessments to monitor for any side effects or health changes during the study.

The study team will record any treatment-emergent adverse events (side effects that appear after starting treatment), serious adverse events, and adverse events of special interest.

5 Contraception Requirements

If you are able to have children, you must use effective contraception methods throughout the study period, consistent with local regulations for clinical trial participants.

6 Study Completion

The study is expected to run until July 2025, with final results analyzed by August 2026.

After completing the treatment period, there may be a follow-up period to monitor your health after stopping the study medication.

Who Can Join the Study?

You must have a confirmed diagnosis of Graves’ disease with active hyperthyroidism (overactive thyroid that produces too much hormone)
You may or may not have active Graves’ orbitopathy (eye problems related to Graves’ disease)
Some participants will need to have active eye problems with a clinical activity score (CAS) of 3 or higher in their most affected eye
These eye problems must include at least one of the following:
- Lid retraction of 2 mm or more (eyelid pulled back too far)
- Moderate or severe soft tissue involvement (swelling around the eyes)
- Proptosis of 2 mm or more (bulging eyes)
- Diplopia (double vision) that occurs occasionally or all the time
You must be willing to use contraception methods that comply with local regulations for clinical trial participants

Who Cannot Join the Study?

You cannot participate if you have had your thyroid gland removed (total or partial thyroidectomy).
You cannot participate if you’ve received radioactive iodine treatment (a treatment that uses radiation to damage thyroid cells).
You cannot participate if you’re pregnant, breastfeeding, or planning to become pregnant during the study.
You cannot participate if you have serious liver problems or liver disease.
You cannot participate if you have serious kidney problems or kidney disease.
You cannot participate if you have active or serious infections.
You cannot participate if you have a history of blood clots.
You cannot participate if you have any other serious medical conditions that the study doctor thinks could make it unsafe for you to participate.
You cannot participate if you’re currently using certain medications that might interact with the study drug.
You cannot participate if you have participated in another clinical trial within the past 30 days.
You cannot participate if you have allergies to the study drug or similar medications.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hospital Universitario De Navarra	Pamplona	Spain

Other Sites

Site Name	City	Country
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone	Palermo	Italy
Central Hospital Of Bolzano	Bolzano	Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Ospedale San Raffaele S.r.l.	Milan	Italy
Hospital Clinic De Barcelona	Barcelona	Spain
Azienda Ospedaliero Universitaria Pisana	Pisa	Italy
General University Hospital Of Larissa	Larissa	Greece
University General Hospital Attikon General Hospital Of West Attica H Agia Varvara	Chaidari	Greece
Unfpxndunyfdssbicpdrf Ezufa Aea	Essen	Germany
Gisfgm Umrbrfjzbh Fpjsngbzn	Frankfurt	Germany
Liuhd Gnarsyj Hvowovht Ol Axirch	Athens	Greece

Trial status

Country	Status	Recruitment Start
Germany	Recruiting	16.07.2025
Greece	Not yet recruiting	16.07.2025
Italy	Recruiting	16.07.2025
Spain	Recruiting	16.07.2025

Trial locations

Investigated drugs:

Rilzabrutinib

Rilzabrutinib is a medication being studied to treat Graves’ disease. It works by targeting and blocking a specific enzyme called Bruton’s tyrosine kinase (BTK) that plays a role in the immune system. By inhibiting this enzyme, rilzabrutinib may help reduce the abnormal immune response that causes the thyroid gland to become overactive in people with Graves’ disease.

Graves’ disease – An autoimmune disorder that causes the thyroid gland to produce excessive thyroid hormones, a condition known as hyperthyroidism. In Graves’ disease, the immune system creates antibodies that stimulate the thyroid gland, leading to overproduction of thyroid hormones such as free thyroxine (FT4) and free triiodothyronine (FT3). Common symptoms include weight loss, increased heart rate, anxiety, tremors, fatigue, and heat intolerance. Some patients may develop eye problems called Graves’ ophthalmopathy, which can cause bulging eyes, double vision, and eye irritation. The disease typically develops gradually and may fluctuate in severity over time.

Trial ID:

2025-521023-75-00

Protocol code:

PDY18663

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Effects and Safety of Rilzabrutinib for Adults with Graves’ Disease

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?