Clinical Trial: Safety and Pharmacokinetics of Alpha-1 MP in Patients With Alpha1-Antitrypsin Deficiency

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase I/II Multicenter, Open-label Trial to Evaluate the Safety and Pharmacokinetics of Alpha-1 MP in Patients With Alpha1-Antitrypsin Deficiency

Brief Summary: This study is a multicenter, open-label trial to evaluate the safety and pharmacokinetics of weekly intravenous infusions of 60 mg/kg of Alpha-1 MP for 8 weeks.

Detailed Summary:

This study is a multicenter, open-label trial to evaluate the safety and pharmacokinetics of weekly intravenous infusions of 60 mg/kg of the investigational drug in subjects with AATD. The trial will be conducted at approximately 5 medical institutions in Japan, aiming to enroll a minimum of 3 adult subjects or more. The trial will consist of a screening period scheduled within 3 weeks before trial entry, an open-label treatment period for 8 weeks, and a PK evaluation period for 1 week. At the Week 9 visit when the PK evaluation period is completed, subjects will be asked whether they would like to participate in an extension trial (GTI1401-OLE). For subjects not intending to participate in the extension trial, the date of follow-up/study completion visit (30 days [4 weeks] after the last dose of the investigational drug) will be arranged. Subjects will participate in this trial for approximately 14 weeks from the start of the screening period through the completion of the trial.

At the screening visit (scheduled within 3 weeks before trial entry), after providing informed consent (agreement based on adequate explanation and understanding of the treatment plan), subjects will be evaluated for eligibility for participation during the screening period. Subjects considered eligible will enter the 8-week treatment period to receive a total of 8 weekly intravenous infusions of 60 mg/kg of Alpha-1 MP. The initial intravenous infusion will be given at the Week 1 (baseline) visit. During the treatment period, subjects will receive weekly intravenous infusions of Alpha-1 MP at the Weeks 1 (baseline), 2, 3, 4, 5, 6, 7, and 8 visits. After the last intravenous infusion of Alpha-1 MP at the Week 8 visit, subjects will enter the 1-week PK evaluation period. During this PK evaluation period, subjects will visit the study center to undergo blood sampling for PK evaluation at the PK1 vis
Sponsor: Grifols Therapeutics Inc.

Current Primary Outcome: Safety of 60 mg/kg Alpha-1 MP assessed by Adverse events, ADRs, serious AEs (SAEs), discontinuations due to AEs or SAEs, and COPD exacerbations [ Time Frame: Baseline to 8 weeks ]

• Adverse events, ADRs, serious AEs (SAEs), discontinuations due to AEs or SAEs, and COPD exacerbations


Original Primary Outcome: Same as current

Current Secondary Outcome: Trough level of total alpha1-PI for weekly IV infusions of 60 mg/kg Alpha-1 MP [ Time Frame: Weeks 1, 7, and 8 (prior to the infusions of Alpha-1 MP) ]

All samples for measurement of alpha1-PI concentration will be analyzed using an antigenic content assay that is validated according to current regulatory and industry expectations


Original Secondary Outcome: Same as current

Information By: Grifols Therapeutics Inc.

Dates:
Date Received: August 12, 2016
Date Started: January 2016
Date Completion: March 2017
Last Updated: December 1, 2016
Last Verified: December 2016