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Proteinase Inhibitor

Alpha1-Proteinase Inhibitor for Alpha-1 Antitrypsin Deficiency

Phase 1 & 2
Recruiting
Research Sponsored by Grifols Therapeutics LLC
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Have a diagnosis of congenital Alpha1-antitrypsin deficiency (AATD) with specific allelic combinations
Have a documented pre-Alpha1-Proteinase Inhibitor (PI) augmentation therapy serum alpha-1 antitrypsin (AAT) level below specified thresholds
Must not have
Females who are pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception throughout the study
Use systemic steroids above a stable dose equivalent to 5 mg/day prednisone within the 4 weeks prior to the Week 1 (Baseline) Visit
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Summary

This trial tests if Alpha-1 protein injections are safe and tolerable for people with Alpha1-Antitrypsin Deficiency. The treatment aims to protect their lungs by giving them extra Alpha-1 protein. Alpha-1 proteinase inhibitor, a human-derived blood product, has been used for over 20 years to treat individuals with Alpha1-Antitrypsin Deficiency.

Who is the study for?
This trial is for individuals with Alpha1-Antitrypsin Deficiency (AATD) who have low serum AAT levels and specific lung function measurements. Participants can be new or current on Alpha1-PI therapy but must stop it before the study starts. Pregnant women, those not using effective contraception, people with severe allergies to plasma-derived treatments, recent lung surgery patients, or those with certain health conditions are excluded.
What is being tested?
The study tests two doses of a medication called Alpha-1 15%, given as a subcutaneous infusion in participants with AATD. The first dose is administered once and then weekly over eight weeks to evaluate safety and how the body processes the drug.
What are the potential side effects?
While specific side effects aren't listed here, common ones for similar treatments may include injection site reactions, possible allergic responses especially in those sensitive to plasma products, headaches, fatigue, and flu-like symptoms.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with Alpha1-antitrypsin deficiency.
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My alpha-1 antitrypsin levels are below the required threshold.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am not pregnant, breastfeeding, and if able to bear children, I agree to use effective birth control during the study.
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I haven't increased my steroid dose above 5 mg/day prednisone in the last 4 weeks.
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I have had a serious worsening of my COPD in the last 4 weeks.
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I have had a lung or liver transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

6Treatment groups
Experimental Treatment
Group I: Cohort 2: Treatment Period 2 (Alpha-1 15%, 180 mg/kg)Experimental Treatment1 Intervention
Following treatment period 1 and single-dose data evaluation phase, participants in Cohort 2 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 180 mg/kg, for 8 weekly SC infusions.
Group II: Cohort 2: Treatment Period 1 (Alpha-1 15%, 180 mg/kg)Experimental Treatment1 Intervention
Participants will receive Alpha-1 15% 180 mg/kg, single weekly SC infusion in treatment-period 1 (Single-Dose) at Week 1.
Group III: Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor 120 mg/kg)Experimental Treatment1 Intervention
Following treatment period 1, participants in Cohort 2 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation phase. During the single-dose data evaluation phase, Liquid Alpha1-PI 120 mg/kg, weekly IV Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose.
Group IV: Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg)Experimental Treatment1 Intervention
Following treatment period 1 and single-dose data evaluation period, participants in Cohort 1 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 72 mg/kg, for 8 weekly SC infusions.
Group V: Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg)Experimental Treatment1 Intervention
Participants will receive Alpha-1 15% 72 mg/kg, single weekly subcutaneous (SC) infusion in treatment-period 1 (Single-Dose) at Week 1.
Group VI: Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibitor 60 mg/kg)Experimental Treatment1 Intervention
Following treatment period 1, participants in Cohort 1 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation period. During the single-dose data evaluation phase, Liquid Alpha1- Proteinase Inhibitor (PI) 60 mg/kg, weekly intravenous (IV) Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose.

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Augmentation therapy for Alpha-1 Antitrypsin Deficiency (AATD) involves administering alpha-1 proteinase inhibitor (A1-PI) to raise the levels of this protective protein in the blood and lungs. This therapy helps shield lung tissue from damage by neutrophil elastase, an enzyme that can degrade proteins and cause severe lung damage in AATD patients. By boosting A1-PI levels, augmentation therapy aims to slow lung disease progression, which is vital for improving the quality of life and reducing exacerbations and hospitalizations in AATD patients.
Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease.The intrapulmonary half-life and safety of aerosolized alpha1-protease inhibitor in normal volunteers.

Find a Location

Who is running the clinical trial?

Grifols Therapeutics LLCLead Sponsor
57 Previous Clinical Trials
5,792 Total Patients Enrolled

Media Library

Alpha-1 15% (Proteinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04722887 — Phase 1 & 2
Alpha-1 Antitrypsin Deficiency Research Study Groups: Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor 120 mg/kg), Cohort 2: Treatment Period 2 (Alpha-1 15%, 180 mg/kg), Cohort 2: Treatment Period 1 (Alpha-1 15%, 180 mg/kg), Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg), Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibitor 60 mg/kg), Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg)
Alpha-1 15% (Proteinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04722887 — Phase 1 & 2
~2 spots leftby Mar 2025