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Ibrutinib

Phase 2

Waldenstrom Macroglobulinemia | Small molecule | Other |Amgen Inc.|Last Updated: Apr 4, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment99
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04263480Efficacy and Safety of Carfilzomib in Combination With Ibrutinib vs Ibrutinib in Waldenström's MacroglobulinemiaPHASE2 ACTIVE NOT_RECRUITING 99Aug 18, 2021Aug 1, 2028Apr 4, 202520 Austria, Germany +1
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Study Endpoints
Primary Endpoints
CR/VGPR
12 months

Primary endpoint is the rate of CR or VGPR 12 months after the start of treatment using the response criteria updated at the Sixth IWWM (CR/VGPR).

Secondary Endpoints
Response rate
12/ 24 months
Best response
12 months
Time to best response
12 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Arm A: Carfilzomib + IbrutinibEXPERIMENTALPatients will be treated with Ibrutinib until evidence of progressive disease or no longer tolerated. Patients will receive in addition Carfilzomib for two years.
Arm B: IbrutinibACTIVE_COMPARATORPatients will be treated with Ibrutinib until evidence of progressive disease or no longer tolerated.
Interventions
NameTypeDescription
Carfilzomib + IbrutinibDRUGCarfilzomib: Cycle 1, day 1: 20 mg/m² i.v. Cycle 1, day 8, day 15: 70 mg/m² i.v. Cycle 2 - 12, day 1, day 8, day 15: 70 mg/m² i.v. Cycle 13 - 24, day 1, day 15: 70 mg/m² i.v. Ibrutinib: 420 mg p.o daily until disease progression or non-tolerable toxicities
IbrutinibDRUGIbrutinib: 420 mg p.o daily until disease progression or non-tolerable toxicities
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: Each patient must meet all of the following inclusion criteria to be enrolled in this study: * Proven clinicopathological diagnosis of WM as defined by consensus panel one of the Second International Workshop on WM. Histopathology has to occur before randomization within the la...

Countries:AustriaGermanyGreece
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT04263480studyFirstPostDate: changed