NCT04722887

A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency

A Multi-Center, Single-Dose and Repeat-Dose Over Eight Weeks, Sequential Cohort Study to Evaluate Safety and Tolerability as Well as Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% Administered Subcutaneously in Subjects With Alpha1-Antitrypsin Deficiency

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Grifols Therapeutics LLC · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC infusions over 8 weeks in participants with Alpha1-Antitrypsin Deficiency (AATD).

Conditions

• Alpha1-Antitrypsin Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	Alpha-1 15%	Alpha1-Proteinase Inhibitor (Human), 15%, Subcutaneous infusion
BIOLOGICAL	Liquid Alpha1-Proteinase Inhibitor (Human)	Intravenous infusion

Timeline

• Start date: 2021-08-13
• Primary completion: 2025-08-01
• Completion: 2025-08-01
• First posted: 2021-01-25
• Last updated: 2025-12-12

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study