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Completed

NCT Number: NCT04722887

A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency

The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC infusions over 8 weeks in participants with Alpha1-Antitrypsin Deficiency (AATD).

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Key information

Conditions

Alpha1-Antitrypsin Deficiency Chronic Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Disease Attributes Emphysema Genetic Diseases, Inborn Liver Diseases Lung Diseases Lung Diseases, Obstructive Pathologic Processes Pathological Conditions, Signs and Symptoms Pulmonary Disease, Chronic Obstructive Pulmonary Emphysema Respiratory Tract Diseases Subcutaneous Emphysema alpha 1-Antitrypsin Deficiency

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

UCLA Medical Center, Los Angeles, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Have a diagnosis of congenital Alpha1-antitrypsin deficiency (AATD) with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles (subjects with "at-risk" alleles must be individually evaluated for eligibility by the Medical Monitor).
  • Have a documented pre-Alpha1-Proteinase Inhibitor (PI) augmentation therapy serum alpha-1 antitrypsin (AAT) level <11 micrometer (μM) (80 milligrams per decilitre (mg/dL) if measured by radial immunodiffusion or 50 mg/dL if measured by nephelometry).
  • Subjects may be naïve to Alpha1-PI augmentation therapy or may be currently receiving Alpha1-PI augmentation therapy or received Alpha1-PI augmentation therapy within the past. If the subject is currently receiving Alpha1-PI augmentation therapy of any kind, he/she must be willing to discontinue that treatment for at least 25 days prior to the Week 1 (Baseline) Visit and remain off any kind of Alpha1-PI treatment, other than the IPs for this study, while participating in the study.
  • At the Screening Visit, have a post-bronchodilator forced expiratory volume (FEV1) ≥30% and <80% of predicted and FEV1/forced vital capacity (FVC) <70% (Global Initiative for Chronic Obstructive Lung Disease (GOLD) stage II or III).

Exclusion criteria

  • 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.
  • Have any lung surgery during the past 2 years (excluding lung biopsy).
  • Have severe concomitant disease (example, 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.
  • Have smoked during the past 6 months or a positive urine cotinine test at the Screening Visit that is due to smoking.
  • Participate in another Investigational product (IP) study within one month prior to the Week 1 (Baseline) Visit.
  • 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. It is recommended to maintain the same dose throughout the study.
  • Use systemic or aerosolized antibiotics for a chronic COPD exacerbation within the 4 weeks prior to the Week 1 (Baseline) Visit.
  • Have known selective or severe Immunoglobulin A (IgA) deficiency.

Treatment and study plan

Alpha-1 15%

Biological

Alpha1-Proteinase Inhibitor (Human), 15%, Subcutaneous infusion

Liquid Alpha1-Proteinase Inhibitor (Human)

Biological

Intravenous infusion

Other names: Prolastin®-C Liquid

Primary outcomes

  1. Number of Participants With Adverse Events (AEs)

    Time frame: Up to 668 days

  2. Number of Participants With Suspected Adverse Drug Reactions (ADRs)

    Time frame: Up to 668 days

  3. Number of Participants With Infusion Site Reactions

    Time frame: Up to 668 days

  4. Number of Participants With Serious Adverse Events (SAEs)

    Time frame: Up to 668 days

  5. Number of Participants With AEs and SAEs Leading to Discontinuation

    Time frame: Up to 668 days

  6. Number of Participants With Chronic Obstructive Pulmonary Disease (COPD) Exacerbations

    Time frame: Up to 668 days

  7. Number of Participants With Clinically Significant Abnormalities in Vital Signs (Heart Rate, Blood Pressure, Respiratory Rate, and Temperature)

    Time frame: Up to 668 days

  8. Change from Baseline in Forced Expiratory Volume in 1 Second (FEV1)

    Time frame: Up to 668 days

  9. Change from Baseline in Forced Vital Capacity (FVC)

    Time frame: Up to 668 days

  10. Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters (Chemistry, Hematology, Urinalysis)

    Time frame: Up to 668 days

  11. Immunogenicity: Number of Participants With Alpha1-PI Antibodies

    Time frame: Treatment Period 1- Single-Dose Week 1; Treatment Period 2- Repeat-Dose Weeks 1 and 9

Sponsors and collaborators

Lead sponsor

Grifols Therapeutics LLC

Industry

Registry information

Official study title

A Multi-Center, Single-Dose and Repeat-Dose Over Eight Weeks, Sequential Cohort Study to Evaluate Safety and Tolerability as Well as Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% Administered Subcutaneously in Subjects With Alpha1-Antitrypsin Deficiency

Important dates

Study start
2021
Primary completion
2025
Study completion
2025
First posted
Jan 25, 2021
Registry last updated
Dec 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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