What is Alpha1-Proteinase Inhibitor studying for Alpha-1 Antitrypsin Deficiency?

Alpha1-Proteinase Inhibitor is being studied to compare two different weekly doses given under the skin with standard intravenous doses in adults with Alpha-1 Antitrypsin Deficiency. Researchers want to understand how the body processes these doses and evaluate their safety and tolerability. The study is sponsored by Grifols Therapeutics LLC.

Who may be eligible for this Alpha-1 Antitrypsin Deficiency clinical trial?

Adults aged 18 to 80 years with a confirmed diagnosis of Alpha-1 Antitrypsin Deficiency and specific genetic types may be eligible. Both men and women can participate if they meet lung function and treatment history criteria. Participants should not have had recent severe lung flare-ups or recent smoking history. Please review eligibility criteria for more details.

Is there any cost to participate in this Alpha-1 Antitrypsin Deficiency clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan for any travel or accommodation costs that may arise.

Actively Recruiting

Phase 3

Age: 18Years - 80Years

All Genders

ID07555483

Study of Weekly Subcutaneous Alpha1-Proteinase Inhibitor Doses Compared to Standard Intravenous Treatment in Adults With Alpha1-Antitrypsin Deficiency

Led by Grifols Therapeutics LLC · Updated on 2026-06-01

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating different weekly doses of Alpha1-Proteinase Inhibitor Alpha1-PI administered under the skin compared to the standard doses given through an intravenous IV infusion in adults with Alpha-1 Antitrypsin Deficiency AATD. This Phase 3 study aims to understand how the body processes these doses and to assess their safety and tolerability. Participants will be randomly assigned to treatment groups, and both participants and study doctors will know which treatment is given. The study involves two treatment arms. One group receives 8 weeks of IV treatment with 60 mgkgweek of Liquid Alpha1-PI followed by 8 weeks of subcutaneous SC treatment with 90 mgkgweek of Alpha-1 15. The other group receives 8 weeks of IV treatment with 120 mgkgweek of Liquid Alpha1-PI followed by 8 weeks of SC treatment with 180 mgkgweek of Alpha-1 15. These treatments are given weekly, and the study compares the effects of these doses over a total of 16 weeks. Participants will attend regular visits for monitoring and assessments during both the IV and SC treatment periods. Researchers will measure how much Alpha1-PI remains in the body over the weekly dosing interval, along with safety and tolerability. Blood tests, lung function tests, and other evaluations will be conducted throughout the study. The total participation duration is about 16 weeks, during which safety and effectiveness data will be collected and analyzed.

CONDITIONS

Brief Title

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)

Who Can Participate

Age: 18Years - 80Years

All Genders

Eligibility Criteria

You may qualify if you...

Adults aged 18 to 80 years
Diagnosed with congenital Alpha-1 Antitrypsin Deficiency with allelic combinations ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles
May be new to or currently receiving alpha1-PI augmentation therapy
Documented total alpha1-PI serum level less than 11 μM (80 mg/dL by radial immunodiffusion or 50 mg/dL by nephelometry)
Post-bronchodilator FEV1 ≥ 25% and < 80% predicted, and FEV1/FVC < 70% (GOLD stage II-III, some stage IV)
Willing to stop any current alpha1-PI treatment at baseline and use only study treatments
Able and willing to provide informed consent indicating understanding and agreement to participate

You will not qualify if you...

Moderate or severe COPD exacerbation within 4 weeks before baseline visit
History of lung or liver transplant or on transplant waiting list
Lung surgery in the past year (except lung biopsy)
Elevated liver enzymes ≥ 2.5 times upper limit of normal at screening
Severe diseases such as congestive heart failure, pulmonary fibrosis, malignant disease (except some skin cancers), or hypersensitivity pneumonitis
Pregnant, breastfeeding, or unwilling to use highly effective contraception if of child-bearing potential
Known infection or symptoms of Hepatitis B, Hepatitis C, or HIV
Smoking or use of electronic/vapor cigarettes in past 6 months
Recent participation in other investigational products or extended half-life AAT products
History of anaphylaxis or severe reaction to plasma-derived alpha1-PI or blood products
Use of systemic steroids above stable low dose or recent antibiotics for COPD exacerbation
Known severe Immunoglobulin A deficiency
Medical or compliance issues that may increase safety risk or limit ability to attend study visits

Research Team

Beatriz Garcia Castro

Elsa Mondou

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