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Advate

Phase 3

Hemophilia | Small molecule | Hematology |Baxter International Inc.|Last Updated: Apr 1, 2020

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment16
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02306694Prospective Biomarkers of Bone Metabolism in Hemophilia APHASE3 COMPLETED 16Dec 1, 2014Apr 16, 2018Apr 1, 20201 United States
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Study Endpoints
Primary Endpoints
Bone Biomarker Density (BMD)
5 days

BMD was measured as Z-scores/T-scores using Dual-Energy X-ray Absorptiometry (DEXA) scanning; specifically looking at Spine, Hip/Neck, and Hip total scores. BMD Z-scores compare what would be expected in someone your age and body size. A Z-score, is a unit of standard deviation, where above 0 would indicate the bones are more dense than expected, while a Z-score below 0 would indicate the bones were less dense.

Joint Health
5 days

Hemophilia Joint Health Score (HJHS); with a higher score representing worst outcomes, scores could range from 0 (no problems) to a max score of 120 (severe problems).

Quality of Life Using the VAS and EQ-5D-3L
5 days

Visual Analog Scale (VAS) via the EQ-5D-3L was used to report participants self-rated health. EQ-5D-3L total score ranges from 5 (no problems) to 15 (significant problems). VAS scores could range from 0 (worst health ever) to 100 (best health ever).

Plasma Cytokine Concentration Differences From 0-hour to 24-hour
24 hours

cytokines were measured using ELISA/magnetic bead multiplex kits. We calculated concentration change from hour 0 to hour 24. Cytokines: FGF, C-Terminal telopeptide (CTX-1), Dickkopf WNT signaling pathway inhibitor 1(DKK1), Eotaxin, fibroblast growth factor 23(FGF23), interferon gamma, interleukin 13, interleukin 1 beta, interleukin receptor 1 antagonist, interleukin 2, interleukin 4, interleukin 6, interleukin 17, interleukin 8, interleukin 9 Insulin, interferon gamma induced protein 10 (IP10), Leptin, monocyte chemoattractant protein1, monocyte chemoattractant protein1, macrophage inflammatory protein 1a (MIP1a), osteoclasts, Osteoprotegerin, osteopontin, platelet derived growth factors, parathyroid horomone, Recepter activator of nuclear factor kappa-B ligand (RANKL), chemokine ligand 5, sclerostin, transforming growth factor-beta 1, transforming growth factor beta 2, transforming growth factor beta 3, tumor necrosis factor alpha, vascular endothelial growth factor, MIP1b.

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Open labelEXPERIMENTALEveryone receives Advate (antihemophilic factor) on Day 1 and 3.
Interventions
NameTypeDescription
AdvateDRUGPatients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.
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Eligibility Criteria
Age Range16 Years — 85 Years
SexMALE
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Males with a diagnosis of hemophilia A with a historic baseline FVIII level ≤ 2%. 2. Age \> 16 years old 3. Currently using ADVATE as FVIII replacement therapy Exclusion Criteria: 1. Subject or guardian is unwilling or unable to give written informed consent and/or assent 2...

Countries:United States
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