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Plasma-derived AT-III

Phase 2

Antithrombin III Deficiency | Small molecule | Other |Grifols, S.A.|Last Updated: Aug 25, 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
UNCONTROLLEDDMC
Total Trials1
Total Enrollment30
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00319228Safety, Pharmacokinetics and Efficacy of an ATIII ConcentratePHASE2 ACTIVE NOT_RECRUITING 30 — — Jan 1, 2006Mar 1, 2029Aug 25, 2023 - —
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Study Endpoints
Primary Endpoints
The primary objectives of this clinical study are to:
2 years
Assess the pharmacokinetic (PK) profile of AT-III in congenital AT-III deficient patients
1 year
To measure the in vivo recovery and half-life of AT-III.
1 year
To assess the clinical safety and tolerability of AT-III-DAF/DI.
1 year
Secondary Endpoints
To assess clinical efficacy by preventing thromboembolic or thrombotic events (prophylaxis) in individuals with congenital AT-III deficiency who are undergoing surgical procedures or who are pregnant and undergoing parturition.
1 year
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposePREVENTION
Treatment Arms
ArmTypeDescription
Antithrombin IIIEXPERIMENTAL -
Interventions
NameTypeDescription
Plasma-derived AT-III concentrateDRUGSegment I: A single dose IV infusion of 50 IU/kg of ATIII-DAF/DI will be administered to each patient. Segment II: A single dose or multiple doses depending on the subject's ATIII plasma levels and patient's specific treatment plan.
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo

Inclusion Criteria: * Congenital ATIII deficiency documented by determination of plasma levels of ATIII off all therapies. Specifically, the baseline levels of ATIII activity should be equal to or less than 60%. * Age \>12 years with a body weight of no less than 30 kg. * Have not participated in a...

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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT00319228primaryCompletionDate: changed
LOWMay 24, 2026NCT00319228studyFirstPostDate: changed