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Rilzabrutinib dose 1

Phase 2

Graves' Disease | Small molecule | Endocrine |Sanofi|Last Updated: Jul 7, 2026

Target and mechanism

Molecular targetBTK
Target classInhibitor
ModalitySmall molecule

Also known as rilzabrutinib, Rilzabrutinib

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLED
Total Trials1
Total Enrollment30

FDA Designations

ORPHAN_DRUGFAST_TRACK

Clinical trial landscape

Rilzabrutinib dose 1 · 1 trial · 1 indication

Phase 2 1
NCT06984627A Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' DiseaseGraves' Disease
RECRUITING30 Analytics
PHASE2RECRUITING
A Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' Disease
Graves' DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Percent change from baseline in FT4 levels
At Week16

Free thyroxine

Secondary Endpoints

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
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Rilzabrutinib dose 1EXPERIMENTALRilzabrutinib
Rilzabrutinib dose 2EXPERIMENTALRilzabrutinib

Interventions

NameTypeDescription
Rilzabrutinib dose 1DRUGPharmaceutical form:Tablet-Route of administration:Oral
Rilzabrutinib dose 2DRUGPharmaceutical form:Tablet-Route of administration:Oral
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites15

Inclusion Criteria: * 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 s...

Countries:CanadaGermanyItalySpainUnited Kingdom
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Recent Changes (Last 90 Days)

LOWJul 7, 2026NCT06984627lastUpdatePostDate: changed
LOWJul 7, 2026NCT06984627lastUpdatePostDate: changed

Frequently asked questions about Rilzabrutinib dose 1

What is Rilzabrutinib used for?

Rilzabrutinib is an investigational small molecule being studied for immune thrombocytopenia (ITP), atopic dermatitis, warm autoimmune hemolytic anemia, chronic spontaneous urticaria, and hepatic function abnormalities. It is also evaluated in healthy volunteers. The drug is not approved and remains in clinical development.

What does Rilzabrutinib target?

Rilzabrutinib is a kinase inhibitor, belonging to the -tinib class of drugs. It is designed to inhibit Bruton's tyrosine kinase (BTK), a key enzyme in B-cell and mast cell signaling pathways. This mechanism is being investigated for autoimmune and allergic conditions.

Who makes Rilzabrutinib?

Rilzabrutinib is developed by Sanofi, a global biopharmaceutical company listed on the stock exchange under the ticker SNY. Sanofi is conducting clinical trials to evaluate the drug's safety and efficacy across multiple indications.

What phase is Rilzabrutinib in?

Rilzabrutinib is in Phase 2 and Phase 3 clinical trials. It has completed Phase 2 studies and is currently in Phase 3 trials for immune thrombocytopenia. The drug has received orphan drug and fast track designations from the FDA, but it is not yet approved.

What clinical trials is Rilzabrutinib in?

Rilzabrutinib is being studied in several trials, including NCT03395210 (Phase 2, completed, ITP), NCT04562766 (Phase 3, active, ITP), NCT05018806 (Phase 2, completed, atopic dermatitis), and NCT07216079 (Phase 3, active, ITP in Japan). These trials are randomized, double-blind, and placebo-controlled.

Is Rilzabrutinib the same as Rilzabrutinib dose 1?

Yes, Rilzabrutinib is also known as Rilzabrutinib dose 1. This alternative name may be used in clinical trial documentation or dosing protocols. Both names refer to the same investigational drug developed by Sanofi.