Study of Alpha1-Proteinase Inhibitor for Alpha-1 Antitrypsin Deficiency
This study is testing two different weekly doses of Alpha1-Proteinase Inhibitor (Alpha1-PI) given as an injection under the skin (subcutaneous) compared to the standard way it's given through a vein (intravenous). It's for adults aged 18 to 80 with Alpha-1 Antitrypsin Deficiency (AATD), specifically those with certain genetic types like ZZ or SZ. The main goals are to see how your body handles the different doses of Alpha1-PI and to check how safe and well-tolerated they are. The study aims to understand if the subcutaneous doses work as well as the intravenous doses. The current recruitment status is unclear.
- Study design
- This is an interventional study with an estimated 40 participants. You will be randomly assigned to a treatment group, and both you and the study doctors will know which treatment you are receiving (open-label).
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary endpoint is measured from Week 1 to Week 16, suggesting follow-up during this period.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT07555483
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)
Recruiting
PHASE3Ages 18–80InterventionalTreatmentGrifols Therapeutics LLC
~40 participants
Updated 2026-08-11 on ClinicalTrials.gov
What's tested:Alpha-1 15%Liquid Alpha1-PI
At a glance
Recruiting sites
7 of 22 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
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
Measured over Week 1 to Week 16
Conditions
Where it's being run
22 sites across 17 statesSpain3
Arizona2
Florida2
Portugal2
Alabama1
California1
Minnesota1
Ohio1
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles (patients with "at-risk" alleles must be individually evaluated for eligibility by the Medical Monitor). If the genotype has yet to be documented, a blood draw for genotyping (i.e., allelic discrimination) and phenotyping will be obtained at the Screening Visit.
Participants may be naïve to alpha1-PI augmentation therapy or may be currently receiving alpha1-PI augmentation therapy or received alpha1-PI augmentation therapy in the past. If the total alpha1-PI serum (alpha-1 antitrypsin \[AAT\]) level has yet to be documented as in a treatment-naïve patient, a blood draw for total alpha1-PI serum level will be obtained at the Screening Visit. For participants currently receiving alpha1-PI augmentation, a pre-alpha1-PI augmentation AAT level must be documented in the participant's medical history/records.
All participants must have a documented total alpha1-PI serum level \<11 μM (80mg/dL if measured by radial immunodiffusion or 50 mg/dL if measured by nephelometry) which is documented pre-alpha1-PI augmentation for participants receiving AAT augmentation.
At the Screening Visit, have post-bronchodilator Forced Expiratory Volume in 1 second (FEV₁) ≥25% and \<80% predicted of predicted FEV₁/Forced Vital Capacity (FVC) \<70% (Global Initiative for Chronic Obstructive Lung Disease \[GOLD\] stage II-III, and some individuals are GOLD stage IV).
If the participant has received alpha1-PI augmentation therapy of any kind, he/she must be willing to discontinue that treatment at the Week 1 (Baseline) Visit and remain off any kind of alpha1-PI treatment, other than the IPs of this study, while participating in the study.
Willing and able to provide written informed consent indicating that they understand the purpose of, and procedures required for the study and are willing to participate in it.
Exclusion
Have had a moderate or severe chronic obstructive pulmonary disease (COPD) exacerbation during the 4 weeks before the Week 1 (Baseline) Visit.
Have history of lung or liver transplant or on transplantation waiting list.
Have any lung surgery during the past 1 year (excluding lung biopsy).
At screening, have elevated liver enzymes (aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\], and alkaline phosphatase \[ALP\]) ≥ 2.5 times the upper limit of normal (ULN).
Have severe concomitant disease (e.g., congestive heart failure, clinically significant pulmonary fibrosis, malignant disease \[except for skin cancers other than melanoma\], history of acute hypersensitivity pneumonitis reaction, or current chronic hypersensitivity pneumonitis).
Females who are pregnant, breastfeeding or, if of child-bearing potential†, unwilling to practice a highly effective method of contraception (oral, injectable, or implanted hormonal methods of contraception, placement of an intrauterine device (IUD) or intrauterine system (IUS), condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence\*) throughout the study. †Women of childbearing potential are defined as premenopausal and not surgically sterile, post tubal ligation, nor documented as infertile due to a concurrent medical condition.
Have known previous infection with or clinical signs and symptoms consistent with current Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), or Human Immunodeficiency Virus (HIV) infection.
Have smoked during the past 6 months (this includes electronic/vapor cigarettes) or a positive urine cotinine test at the Screening Visit that is due to smoking.
Received IP in another study within 30 days prior to the Week 1 (Baseline) Visit or received any recombinant human AAT-Fc fusion protein (e.g., INBRX-101) or other extended half-life AAT products within 5 half-lives of the product relative to the Screening Visit date.
Have history of anaphylaxis or severe systemic response to any plasma-derived alpha1- PI preparation or other blood product(s).
Use systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 4 weeks prior to the Week 1 (Baseline) Visit (Note: inhaled steroids are not considered systemic steroids). It is recommended to maintain the same dose throughout the study.
Use systemic or aerosolized antibiotics for a COPD exacerbation within the 4 weeks prior to the Week 1 (Baseline) Visit.
Have known selective or severe Immunoglobulin A (IgA) deficiency based on prior medical records.
In the opinion of the Investigator, the participant may have compliance problems or any medical condition that may place them at safety risk with the protocol and the procedures of the protocol, or because of unstable health be unable to come to the study site for in-person clinic visits required by the protocol.
What this trial measures
- 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 levelsWeek 1 to Week 16