A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)

Conditions

• Alpha 1 Antitrypsin Deficiency

Eligibility Criteria

Sex

ALL

Age

18 Years - 80 Years

Healthy Volunteers

Not accepted

Interventions

• Alpha-1 15% — DRUG
  Alpha1-Proteinase Inhibitor Subcutaneous (Human), 15%
• Liquid Alpha1-PI — OTHER
  Liquid Alpha1-Proteinase Inhibitor (Human) for Intravenous infusion

Study Details

This study is designed to compare two different weekly doses of a medicine called Alpha1-Proteinase Inhibitor given by injection under the skin with the standard doses of the same medicine given through a vein. Adults with Alpha-1 Antitrypsin Deficiency will take part. Participants will be randomly assigned to one of the treatment groups, and both the study doctors and participants will know which treatment is being given. The main goals of the study are to understand how the body processes the medicine (pharmacokinetics) and to assess how safe and well tolerated the different weekly doses are.

Key Dates

First listed

Apr 29, 2026

Start date

May 5, 2026

Status verified

Aug 2026

Primary completion

Sep 30, 2027

Completion

Sep 30, 2027

Study Design

Enrollment

40 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Treatment Arm 1 (60 mg/kg/week of Liquid Alpha1-PI followed by 90 mg/kg/week of Alpha-1 15%)
  8 weeks of IV treatment with 60 mg/kg/week Liquid Alpha1-PI followed by 8 weeks of SC treatment with 90 mg/kg/week Alpha-1 15%
• Experimental: Treatment Arm 2 (120 mg/kg/week of Liquid Alpha1-PI followed by 180 mg/kg/week of Alpha-1 15%)
  8 weeks of IV treatment with 120 mg/kg/week Liquid Alpha1-PI followed by 8 weeks of SC treatment with 180 mg/kg/week Alpha-1 15%

Primary Outcome Measure

Steady-state AUC of alpha1-PI over the weekly dosing interval (from 0 to 7 days) (AUC0-7 days) in the IV Treatment Period 1 and in the SC Treatment Period 2 for both dose levels [ Time Frame: Week 1 to Week 16 ]

Central Contacts

• Beatriz Garcia Castro
  +(34) 670923669
  [email protected]
• Lydia Martinez Garrido
  +(34) 670929415
  [email protected]

Locations (10)

Find similar trials in Birmingham, AL

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