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)

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Grifols Therapeutics LLC
Study ID
NCT07555483
Phase
PHASE3
Status
Recruiting
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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

Start date
May 5, 2026
Status verified
May 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

Locations (10)

FacilityCityStateZIPSite coordinators
University of Alabama at BirminghamBirminghamAlabama35233
Dianne Freeman
J. Michael Wells, MD (PRINCIPAL_INVESTIGATOR)
Pulmonary AssociatesPhoenixArizona85032
Liyi Fu
Da-Wei Liao, MD (PRINCIPAL_INVESTIGATOR)
Mayo ClinicScottsdaleArizona85259
Ciara Terry
Garrett Ashley, MD (PRINCIPAL_INVESTIGATOR)
UCLALos AngelesCalifornia90095
Tiffany Bina
Igor Barjaktarevic, MD, PhD (PRINCIPAL_INVESTIGATOR)
University of FloridaGainesvilleFlorida32610
Brandi Lattinville
Jorge Lascano, MD (PRINCIPAL_INVESTIGATOR)
University of MiamiMiamiFlorida33136
Tiffany Salcito
Michael Campos, MD (PRINCIPAL_INVESTIGATOR)
Mayo ClinicRochesterMinnesota55905
Sam Fatis
Megan Dulohery-Scrodin, MD (PRINCIPAL_INVESTIGATOR)
Cleveland ClinicClevelandOhio44195
Shauna Hendershot
Umur Hatipoglu, MD (PRINCIPAL_INVESTIGATOR)
Medical University of South CarolinaCharlestonSouth Carolina29425
Gwen Hayden
Charlie Strange, MD (PRINCIPAL_INVESTIGATOR)
UT Southwestern Medical CenterDallasTexas75390
Rhoda Annoh Gordon
Carlos Cardenas, MD (PRINCIPAL_INVESTIGATOR)

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By research site
University of Alabama at Birmingham· Birmingham, ALPulmonary Associates· Phoenix, AZMayo Clinic· Scottsdale, AZUCLA· Los Angeles, CAUniversity of Florida· Gainesville, FLUniversity of Miami· Miami, FL

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