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Granulocyte Colony-Stimulating Factor

Phase 2

Aplastic Anemia | Small molecule | Hematology |Incyte Corporation|Last Updated: May 22, 2026

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 Enrollment1
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06752694Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic AnemiaPHASE2 ACTIVE NOT_RECRUITING 1 — — Sep 25, 2025Oct 15, 2026May 22, 20261 United States
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Study Endpoints
Primary Endpoints
Incidence of grades II-IV acute graft-versus-host disease (GVHD)
At day 100

Will be estimated as simple proportions and informally compared to the historical controls at Fred Hutch. (i.e., estimates presented descriptively, but no formal statistical comparisons will be made).

Secondary Endpoints
Incidence of grade III-IV acute GVHD
At day 100
Incidence of chronic GVHD
At 1 year
Incidence of primary graft failure
At 2 years
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Prevention (conditioning, transplant, GVHD prophylaxis)EXPERIMENTALSee Detailed Description.
Interventions
NameTypeDescription
Biospecimen CollectionPROCEDUREUndergo blood sample collection
Bone Marrow AspirationPROCEDUREUndergo bone marrow biopsy and aspiration
Bone Marrow BiopsyPROCEDUREUndergo bone marrow biopsy and aspiration
Bone Marrow TransplantationPROCEDUREUndergo bone marrow transplant
Computed TomographyPROCEDUREUndergo CT
CyclosporineDRUGGiven PO
FludarabineDRUGGiven IV
Granulocyte Colony-Stimulating FactorDRUGGiven SC
Mycophenolate MofetilDRUGGiven PO
Peripheral Blood Stem Cell TransplantationPROCEDUREUndergo PBSC transplantation
RuxolitinibDRUGGiven PO
SirolimusDRUGGiven PO
Total-Body IrradiationRADIATIONUndergo TBI
Multigated Acquisition ScanPROCEDUREUndergo MUGA
EchocardiographyPROCEDUREUndergo ECHO
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Age \> 40 years or ages 18 - 40 years with Hematopoietic Cell Transplantation - Specific Comorbidity Index (HCT-CI) score \> 3 necessitating a low intensity transplant or determined inability to tolerate antithymocyte globulin (ATG) * Diagnosis of severe acquired aplastic anem...

Countries:United States
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Competitive Landscape -Aplastic Anemia 4 trials
CompanyTickerTrialsLead PhaseDrugs
Pfizer Inc.PFE1 — Anti-human thymocyte immunoglobulin, equine
Novartis AG Sponsored ADRNVS1 — Undisclosed
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Recent Changes (Last 90 Days)
HIGHMay 26, 2026NCT06752694Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 24, 2026NCT06752694studyFirstPostDate: changed