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Liquid Alpha₁-PI

Phase 2

Alpha₁-Antitrypsin Deficiency | Monoclonal antibody | Rare Disease |Grifols, S.A.|Last Updated: Mar 13, 2017

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment32

FDA Designations

No designations recorded

Clinical trial landscape

Liquid Alpha₁-PI · 1 trial · 1 indication

Phase 2 1
NCT02282527A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin DeficiencyAlpha₁-Antitrypsin Deficiency
COMPLETED32 Analytics
PHASE2COMPLETED
A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency
Alpha₁-Antitrypsin DeficiencyUnlock trial analytics

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 to 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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Frequently asked questions about Liquid Alpha₁-PI

What is Liquid Alpha₁-PI used for?

Liquid Alpha₁-PI is an investigational therapy being studied for the treatment of Alpha₁-Antitrypsin Deficiency, a rare genetic condition. It is a liquid formulation of alpha₁-proteinase inhibitor (human) intended to address the deficiency of this protective protein in affected individuals.

Who makes Liquid Alpha₁-PI?

Liquid Alpha₁-PI is being developed by Grifols, S.A., a biopharmaceutical company. Grifols is listed on the stock exchange under the ticker symbol GRFS.

What phase is Liquid Alpha₁-PI in?

Liquid Alpha₁-PI is currently in Phase 2 of clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still being evaluated in clinical trials for safety and efficacy.

What clinical trials is Liquid Alpha₁-PI in?

Liquid Alpha₁-PI has one completed clinical trial, registered as NCT02282527. This Phase 2 study assessed the safety and pharmacokinetics of Liquid Alpha₁-Proteinase Inhibitor (Human) in treating Alpha₁-Antitrypsin Deficiency. The trial enrolled 32 participants in the United States.

How does Liquid Alpha₁-PI work?

Liquid Alpha₁-PI is a monoclonal antibody designed to supplement or replace the deficient alpha₁-proteinase inhibitor in patients with Alpha₁-Antitrypsin Deficiency. By providing this protein, it aims to protect lung tissue from damage caused by uncontrolled protease activity.