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Refametinib

Phase 2

Carcinoma, Hepatocellular | Small molecule | Oncology |Bayer AG|Last Updated: Apr 8, 2021

Success Probability

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Trial Design

UNCONTROLLED
Total Trials2
Total Enrollment30

FDA Designations

No designations recorded

Clinical trial landscape

Refametinib · 3 trials · 2 indications

Phase 2 2Phase 1 1
NCT01915602Refametinib in Combination With Sorafenib in RAS Mutant Hepatocellular Carcinoma (HCC)Carcinoma, Hepatocellular
COMPLETED14 Analytics
NCT01915589Refametinib(BAY86-9766) in RAS Mutant Hepatocellular Carcinoma (HCC)Carcinoma, Hepatocellular
COMPLETED16 Analytics
PHASE2COMPLETED
Refametinib in Combination With Sorafenib in RAS Mutant Hepatocellular Carcinoma (HCC)
Carcinoma, HepatocellularUnlock trial analytics
PHASE2COMPLETED
Refametinib(BAY86-9766) in RAS Mutant Hepatocellular Carcinoma (HCC)
Carcinoma, HepatocellularUnlock trial analytics

Study Endpoints

Primary Endpoints

Objective tumor response according to Modified Response Evaluation Criteria in Solid Tumors (mRECIST) assessed by central radiological review
Approximately 36 months
Plasma concentration of BAY86-9766
Multiple time points up to 6 day
Plasma concentration of M17 and M11
Multiple time points up to 6 day
Plasma concentration of Gemcitabine
Multiple time points up to 6 day
Plasma concentration of dFdU
Multiple time points up to 6 day
Number of adverse events, or abnormal parameters as a measure of safety and tolerability
Up to 1 year

Parameters are laboratory parameters, vital signs, ECG parameters, cardiac function, and parameters of ophthalmologic examinations

Secondary Endpoints

Objective tumor response according to Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 assessed by central radiological review
Approximately 36 months
Objective tumor response according to RECIST 1.1 and mRECIST assessed by investigators
Approximately 36 months
Disease control (central and investigator's assessment)
Approximately 36 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Refametinib and Sorafenib (Nexavar)EXPERIMENTALIn Cycle 1 reduced sorafenib dose (600 mg daily; 200 mg in the morning + 400 mg in the evening) is administered, which is escalated to the standard dose in Cycle 2, if no Hand-foot skin reaction (HFSR), fatigue, or gastrointestinal (GI) toxicities of grade 2 or higher occur. For the purposes of data recording, the treatment period will be divided into 3-week cycles. Patients will continue on treatment until at least one of the following occurs (main criteria): Progressive Disease (PD) {PD as defined by mRECIST criteria or clinical progression \[e.g. Eastern Cooperative Oncology Group performance status (ECOG PS) of ≥3\], treatment may be continued past radiological progression, provided the patient derives clinical benefit as judged by the treating physician.}, Death, Unacceptable toxicity, Subject withdraws consent, Treating physician determines discontinuation of treatment is in the subject's best interest, Substantial non-compliance with the protocol
Refametinib (BAY86-9766)EXPERIMENTALFor purposes of data recording, the treatment period will be divided into 3-week cycles. Patients will continue on treatment until at least one of the following occurs (main criteria): Death Unacceptable toxicity Subject withdraws consent Substantial non-compliance with the protocol Treating physician determines discontinuation of treatment is in the subject's best interest. Radiological progression as determined by RECIST (Version 1.1) or mRECIST criteria or clinical progression (e.g. Eastern Cooperative Oncology group performance status - ECOG PS ≥3) patients may continue to receive study treatment if identified as having continued clinical benefit as judged by the treating physician.
Refametinib (BAY86-9766)+ GemcitabineEXPERIMENTALSingle dose of BAY86-9766 on Cycle 1 Day -17; twice daily dosing every day starting on day -14, start dose 50mg bid ( 30mg or 20mg are possible based on adverse events need) in addition with Gemcitabine intravenous on day 1,8 and 15 1000mg/m2

Interventions

NameTypeDescription
Refametinib (BAY86-9766)DRUGPatients will receive refametinib 50 mg (2x20 mg + 1x10mg capsules or 50 mg tablets) bid
Sorafenib (BAY43-9006)DRUGPatients will receive sorafenib 400 mg (2 x 200 mg tablets) bid.
GemcitabineDRUG -
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites79

Inclusion Criteria: Eligibility criteria for RAS mutation testing * Unresectable or metastatic HCC, confirmed either by histology or clinically according to the American Association for the Study of Liver Disease (AASLD) criteria for cirrhotic patients. For non-cirrhotic patients, histological con...

Countries:United StatesAustriaBelgiumChinaCzechiaFranceGermanyHong KongHungaryIsraelItalyJapanNew ZealandSingaporeSouth KoreaSpainSwitzerlandTaiwanThailandTurkey (Türkiye)United Kingdom
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Frequently asked questions about Refametinib

What is Refametinib used for?

Refametinib is an investigational small molecule being studied for the treatment of hepatocellular carcinoma and other neoplasms. It is being developed by Bayer AG for oncology indications. As of the available data, it is in Phase 2 clinical development and is not yet approved by regulatory authorities.

What does Refametinib target?

Refametinib is a small molecule that targets the MEK pathway, which is involved in cell growth and proliferation. It is being studied in cancers with RAS mutations, particularly in hepatocellular carcinoma. The drug is designed to inhibit this pathway to potentially slow or stop tumor growth.

Who makes Refametinib?

Refametinib is being developed by Bayer AG, a multinational pharmaceutical company. Bayer's stock is traded under the ticker BAYRY on the OTC market. The drug is also known by the code name BAY86-9766 in some clinical trials.

What phase is Refametinib in?

Refametinib is in Phase 2 clinical development. It has completed two clinical trials, including a Phase 1 study and two Phase 2 studies. The drug is investigational and has not received FDA approval. It is being studied for the treatment of hepatocellular carcinoma and other neoplasms.

What clinical trials is Refametinib in?

Refametinib has been studied in three clinical trials, all of which are completed. These include NCT01764828, a Phase 1 study of BAY86-9766 plus gemcitabine in Asian patients with neoplasms; NCT01915589, a Phase 2 study in RAS mutant hepatocellular carcinoma; and NCT01915602, a Phase 2 study of refametinib in combination with sorafenib in RAS mutant hepatocellular carcinoma.

Is Refametinib the same as BAY86-9766?

Yes, Refametinib is also known as BAY86-9766. This alternative name appears in clinical trial titles and descriptions, such as NCT01764828 and NCT01915589. Both names refer to the same investigational drug being developed by Bayer AG for oncology indications.