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Liquid Alpha₁-PI

Phase 2

Alpha₁-Antitrypsin Deficiency | Monoclonal antibody | Other |Grifols, S.A.|Last Updated: Mar 13, 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
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02282527A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin DeficiencyPHASE2 COMPLETED 32Oct 1, 2014Jan 1, 2016Mar 13, 20176 United States
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Study Endpoints
Primary Endpoints
AUC(0-7 Days) Based on Antigenic Content
pre-dose, 0, 15 min, 30 min, 1 hour, 2 hours, 4 hours, 8 hours, 1 day, 2 days, 5 days, 7 days post dose

The primary PK objective of this study was to demonstrate the bioequivalence of Liquid Alpha₁-PI 60 mg/kg to Prolastin-C 60 mg/kg, as measured by AUC from 0 to 7 days (AUC0-7days) using an antigenic content assay of alpha₁-PI, at approximate steady state in subjects with AATD.

Secondary Endpoints
AUC(0-7 Days) Based on Functional Activity
pre-dose, 0, 15 min, 30 min, 1 hour, 2 hours, 4 hours, 8 hours, 1 day, 2 days, 5 days, 7 days post dose
Number of Subjects With Immunogenicity Response
Weeks 1, 9, 17, and 20
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Treatment Sequence 1OTHERSubjects were treated first with Liquid Alpha₁-PI and then treated with Prolastin-C
Treatment Sequence 2OTHERSubjects were treated first with Prolastin-C and then treated with Liquid Alpha₁-PI
Interventions
NameTypeDescription
Liquid Alpha₁-PIBIOLOGICALLiquid Alpha₁-PI, 60 mg/kg, 8 weekly intravenous infusions
Prolastin-CBIOLOGICALProlastin-C, 60 mg/kg, 8 weekly intravenous infusions
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: * Be between 18 and 70 years of age, inclusive * Had a diagnosis of congenital AATD * Had a documented total alpha₁-PI level \< 11 µM. If the total alpha₁-PI level had yet to be documented, a blood draw for total alpha₁-PI level was obtained at the Screening Visit * Had a post-b...

Countries:United States
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