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Anti-thrombin III

Phase 2

ATIII Deficiency | Small molecule | Other |Grifols, S.A.|Last Updated: Nov 25, 2019

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
RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment45
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02103114Anti-thrombin III (ATIII) vs Placebo in Children (<7mo) Undergoing Open Congenital Cardiac SurgeryPHASE2 COMPLETED 45Jun 1, 2014Jul 1, 2016Nov 25, 20191 United States
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Study Endpoints
Primary Endpoints
Difference in the Mean and Standard Deviation (SD) of the Calibrated Automated Thrombography (CAT) Measurements of the Control and ATIII Groups at Time 5 (on Arrival in ICU)
Time 5 (on arrival in ICU)

Evidence of decreased activation of the coagulation and fibrinolytic systems represented by a difference in the mean and Standard Deviation (SD) of the Calibrated Automated Thrombography (CAT) measurements of the control and ATIII groups at Time 5 (on arrival in ICU).

Secondary Endpoints
Difference in the Mean and SD of the Calibrated Automated Thrombography (CAT) Measurements of the Control and ATIII Groups at Times 5-Time 7 (ICU Arrival to Post Operative Day 4)
ICU arrival (Time 5) to Time 7 (Post-operative Day 4)
Difference in the Mean the ATIII (Functional Assay) of the Control and ATIII Groups at T1, T2, T3, T5, T6 and T7
T1, T2, T3, T5, T6 and T7
Difference in the Median of the ATIII (Functional Assay) of the Control and ATIII Groups at T4
T4 (just prior to coming off of CPB)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Anti-thrombin IIIEXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
Anti-thrombin IIIDRUGIntraoperatively- (correcting to 100%) according to the following formula: Units required = ((100%- baseline ATIII level\*%) X body weight)/1.4 * expressed as a % normal level based on functional ATIII assay
PlaceboOTHERNormal saline placebo
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Eligibility Criteria
Age RangeN/A — 7 Months
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * All patients less than 7 months of age going for cardiac surgery that will require cardiopulmonary bypass (CPB) with a documented ATIII level below 70% Exclusion Criteria: * Less than 2.5kg * Known or suspected hereditary ATIII deficiency (family history of venous thrombosis...

Countries:United States
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