Clinical Trial: A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Multi-center, Randomized, Double-blind, Crossover Study to Assess the Safety and Pharmacokinetics of Liquid Alpha₁-Proteinase Inhibitor (Human) Compared to Prolast

Brief Summary: Grifols Therapeutics Inc. conducted a multi-center, randomized, double-blind, crossover study to evaluate the safety, immunogenicity, and pharmacokinetics (PK) of Liquid Alpha₁-PI compared to the currently licensed product, Prolastin-C, in subjects with Alpha₁-Antitrypsin Deficiency (AATD).

Detailed Summary:
Sponsor: Grifols Therapeutics Inc.

Current Primary Outcome: AUC(0-7 Days) Based on Antigenic Content [ Time Frame: 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.


Original Primary Outcome: Area under the concentration vs. time curve (AUC) [ Time Frame: 7 days ]

Current Secondary Outcome:

  • AUC(0-7 Days) Based on Functional Activity [ Time Frame: 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 exploratory 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 (AUC 0-7 days) using a functional activity assay of alpha₁-PI, at approximate steady state in subjects with AATD.
  • Number of Subjects With Immunogenicity Response [ Time Frame: Weeks 1, 9, 17, and 20 ]
    Blood samples for immunogenicity testing were collected at Weeks 1 (Baseline), 9, 17, and 20. Any samples that tested positive for alpha₁-PI antibodies were tested for neutralizing antibodies and antibody titer. Immunogenicity testing was performed using validated assays in a multitiered approach. Samples collected at Week 1 (Baseline) and at Weeks 9 and 20 were tested for immunogenicity while samples collected at Week 17 were to be tested for immunogenicity only if deemed appropriate (eg, unexpected PK profile).


Original Secondary Outcome: Adverse events (AEs) [ Time Frame: 20 weeks ]

Subjects will be monitored for AEs


Information By: Grifols Therapeutics Inc.

Dates:
Date Received: October 31, 2014
Date Started: October 2014
Date Completion:
Last Updated: January 23, 2017
Last Verified: January 2017