Rilzabrutinib for Graves' Disease

Recruiting at 11 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sanofi

Disqualifiers: Non-Graves' hyperthyroidism, Thyroid storm, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 13

Duration: up to 16 weeks

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests rilzabrutinib, a medication that might help treat Graves' disease, a condition where the thyroid produces too much hormone, causing symptoms like weight loss and anxiety. The study aims to evaluate the effectiveness and safety of different doses of rilzabrutinib for individuals with active hyperthyroidism due to Graves' disease, with or without eye problems known as Graves' orbitopathy (characterized by swelling and bulging of the eyes). Individuals diagnosed with Graves' disease and active hyperthyroidism, particularly those who have noticed eye changes like bulging or double vision, might be suitable candidates for this trial. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that rilzabrutinib is generally well-tolerated by patients. In studies involving individuals with immune thrombocytopenia, a condition where the immune system attacks platelets, rilzabrutinib proved safe and did not cause serious side effects.

While these studies do not provide specific information about its use for Graves' disease, an autoimmune disorder affecting the thyroid, the trial's progression to this stage indicates that earlier research deemed rilzabrutinib safe enough for further testing. Although some side effects might occur, they are not expected to be severe. Prospective participants should discuss any concerns or questions with a healthcare provider before joining a trial. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Rilzabrutinib is unique because it targets Bruton’s tyrosine kinase (BTK), a key player in the immune system. Unlike standard treatments for Graves' disease, which often include antithyroid drugs or radioactive iodine therapy, Rilzabrutinib offers a novel mechanism of action by directly inhibiting BTK. This targeted approach may reduce the overactive immune response seen in Graves' disease, potentially offering a more precise and effective treatment with fewer side effects. Researchers are excited about its potential to transform how we manage this autoimmune condition.

What evidence suggests that this trial's treatments could be effective for Graves' disease?

Research shows that rilzabrutinib might help lower thyroid activity in people with Graves' disease. This is important because Graves' disease often causes excessive thyroid activity, leading to health issues. Early studies with rilzabrutinib in other conditions indicate it might help when the immune system is overactive. For instance, it increased platelet counts, which are blood cells that aid in clotting. Although specific data for Graves’ disease is still being collected, these early results suggest rilzabrutinib could be a promising treatment. Participants in this trial will receive one of two different doses of rilzabrutinib to evaluate its effectiveness and safety in treating Graves' disease. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

Adults with confirmed Graves' disease and active hyperthyroidism, which may include those with active eye symptoms (Graves' orbitopathy). Participants must have a clinical activity score of at least 3 for the most affected eye. They should also follow local contraception guidelines if participating in the study.

Inclusion Criteria

I have been diagnosed with Graves' disease and currently have an overactive thyroid.

Contraceptive use by men and women consistent with local regulations regarding methods of contraception for clinical study participants.

I have active Graves' eye disease with a severity score of 3 or higher in one eye.

Exclusion Criteria

My eye condition has not improved with medication.

I have a history of or am at high risk for thyroid storm.

I have had an overactive thyroid not due to Graves' disease.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 4 weeks

Treatment

Participants receive rilzabrutinib dose 1 or dose 2 for Graves' disease

up to 16 weeks

Up to 13 visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Rilzabrutinib

Trial Overview The trial is testing two different doses of Rilzabrutinib to assess its effectiveness and safety in treating Graves' disease. The study includes a screening period, a treatment phase lasting up to 16 weeks, and follows up for an additional four weeks with up to 13 visits.

How Is the Trial Designed?

Group I: Rilzabrutinib dose 2 Experimental Treatment 1 Intervention

Rilzabrutinib

Group II: Rilzabrutinib dose 1 Experimental Treatment 1 Intervention

Rilzabrutinib

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06984627

NCT06984627 | A Study to Investigate the Efficacy and ... 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 ...

2. sanofistudies.com sanofistudies.com/cwout/CW-V9DEXS//gb/en/listing/313075/a-study-to-investigate-26/

A Study to Investigate the Efficacy and Safety 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 ...

3. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-rilzabrutinib-for-adults-with-graves-disease/

Study on the Effects and Safety of Rilzabrutinib for Adults ... The purpose of this research is to determine if rilzabrutinib can reduce thyroid activity in people with Graves' disease. The study is open- ...

4. trial.medpath.com trial.medpath.com/clinical-trial/e77fc76505f71a50/nct06984627-rilzabrutinib-graves-disease-adults

A Study to Investigate the Efficacy and Safety of Rilzabrutinib ... 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 ...

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39844469/

Efficacy and Safety Results With Rilzabrutinib, an Oral ... Nine (35%) patients achieved the primary endpoint. Platelet counts ≥ 50 × 109/L or ≥ 30 × 109/L and doubling from baseline without rescue ...

6. sanofistudies.com sanofistudies.com/cwout/CW-V9DEXS//us/en/listing/313075/a-study-to-investigate-26/

A Study to Investigate the Efficacy and Safety This study investigates the effects and safety of an investigational medication on individuals with Graves' disease, which is an autoimmune ...