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mycophenolate

Phase 2

Graft Versus Host Disease | Small molecule | Immunology |Amgen Inc.|Last Updated: Aug 1, 2017

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment48
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00639717Addition of Etanercept and Extracorporeal Photopheresis (ECP) to Standard Graft-Versus-Host Disease (GVHD) Prophylaxis in Stem Cell TransplantPHASE2 COMPLETED 48Mar 1, 2009Apr 1, 2016Aug 1, 20171 United States
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Study Endpoints
Primary Endpoints
Percentage of Patients Alive at 6 Months
6 months

Overall survival at 6 months

Percentage of Patients Who Experienced Relapse by 6 Months
6 months

Relapse rate at 6 months. Relapse is defined as recurrence of disease.

Secondary Endpoints
The Percentage of Patients That Experienced Graft Versus Host Disease
6 Months
Measured Level of Circulating Plasma Markers After Transplant
100 days
Regulatory T Cell Numbers Post-transplant
180 days
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposePREVENTION
Treatment Arms
ArmTypeDescription
Etanercept and ECPEXPERIMENTALEtanercept and ECP (Extracorporeal Photopheresis) in addition to standard GVHD prevention: Etanercept will be given twice weekly by subcutaneous injection starting on the day of HSCT (Hematopoietic stem cell transplantation) conditioning until 8 weeks post transplant. ECP treatments will begin at once weekly starting at 4 weeks post transplant and continue at less frequent intervals until 6 months post transplant. GVHD prophylaxis will consist of a standard two drug regimen: mycophenolate for 4 weeks and tacrolimus (titrated to a therapeutic level) for 8 weeks, then weaned over 4 months with discontinuation by 6 months post-transplant.
Interventions
NameTypeDescription
stem cell transplantPROCEDUREreduced intensity, matched unrelated donor stem cell transplant
tacrolimus (standard GVHD prophylaxis)DRUGTacrolimus(or cyclosporine when necessary) Tacrolimus will begin on day -3, IV or oral. Target trough level for tacrolimus is 8-12 ng/ml. In the absence of GVHD, tacrolimus tapering will begin on day +56 post transplant
mycophenolate (standard GVHD prophylaxis)DRUGMycophenolate will begin on day 0 at 10 mg/kg/dose (up to 1 gram per dose) every 8 hours orally or intravenously and will continue until day 28.
etanerceptDRUGEtanercept will be given at a dose 0.4 mg/kg (actual weight) up to a maximum dose of 25 mg, subcutaneously, twice weekly from day 0 to day 56 (16 doses)
methoxsalenDRUGMethoxsalen (UVADEX) treatments by Extracorporeal photopheresis (ECP) will be started day +28 post transplant and given weekly. On day +70 post transplant ECP frequency will be given every other week. On day +100 post transplant ECP will be given monthly until day +180 and stopped.
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Eligibility Criteria
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Candidate for unrelated donor (allogeneic) HSCT for hematologic conditions, either malignant or non-malignant. * Donor can be unrelated marrow, blood or cord blood. * Any disease for which unrelated donor transplant is appropriate is eligible except: * Progressive or poorly...

Countries:United States
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