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N-Plate / romiplostim

Phase 2

Myelodysplastic Syndromes | Small molecule | Hematology |Amgen Inc.|Last Updated: Aug 16, 2023

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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment77
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02335268Validation of a Predictive Model of Response to Romiplostim in Patients With IPSS Low or Intermediate-1 Risk MDS and ThrombocytopeniaPHASE2 COMPLETED 77May 21, 2015Jul 1, 2021Aug 16, 202336 France, Germany
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Study Endpoints
Primary Endpoints
Hematologic Improvement of Platelets (HI-P) After 4 Months on Therapy
after 4 months on therapy (week 16)

The primary efficacy endpoint was the rate of HI-P defined as an absolute increase of platelet count to ≥ 30/nL for patients starting at \> 20/nL or an increase of platelets from \< 20/nL to \> 20/nL and by at least 100%, according to IWG 2006 criteria lasting for ≥ 8 weeks after at least 16 Weeks of romiplostim treatment.

Secondary Endpoints
Cumulative Hematologic Improvement
week 16
The Incidence of Disease Progression to Higher Stage MDS or AML
week 16
Increase of Peripheral Blasts During Therapy
week 16
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Group 1EXPERIMENTALStratification into group 1 if Score (Baseline-TPO Level and previous thrombocyte transfusions) are +3 TPO based model to predict subsequent response to romiplostim in MDS patients with IPSS Low/Int-1 according to a model developed by Sekeres et. al./ BJH 2014
Group 2EXPERIMENTALStratification into group 2 if Score (Baseline-TPO Level and previous thrombocyte transfusions) are -1 or -2 TPO based model to predict subsequent response to romiplostim in MDS patients with IPSS Low/Int-1 according to a model developed by Sekeres et. al./ BJH 2014
Group 3EXPERIMENTALStratification into group 3 if Score (Baseline-TPO Level and previous thrombocyte transfusions) are -6 TPO based model to predict subsequent response to romiplostim in MDS patients with IPSS Low/Int-1 according to a model developed by Sekeres et. al./ BJH 2014
Interventions
NameTypeDescription
N-Plate / romiplostimDRUGmedical intervention in 3 patient groups (MDS patients with IPSS Low/Int-1) that are stratified according to their baseline TPO-Level and previous transfusions
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites36

Inclusion Criteria: * Subjects must meet the following inclusion/exclusion criteria to be eligible for the study. * Must understand and voluntarily sign the informed consent form * Age older 18 years at the time of signing the informed consent form * Must be able to adhere to the study visit schedu...

Countries:FranceGermany
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Competitive Landscape -Myelodysplastic Syndromes 76 trials
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