Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)

• Status: Completed
• Trial end date: 2007-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The goal of this trial was to explore the utility of evaluating emphysema progression through CT scans measuring lung density during a 2 year period of weekly infusions of either placebo or human alpha-1-antitrypsin (AAT; Prolastin®). Exacerbation data recorded in patient diaries were also collected. All efficacy data were analyzed for potential use in evaluating Prolastin efficacy in this and other clinical trials.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Grifols Therapeutics Inc.; Grifols Therapeutics LLC
• Treatments: Alpha 1-Antitrypsin; Protease Inhibitors; Protein C Inhibitor

Criteria

Inclusion Criteria:

• Patient with pulmonary emphysema due to severe congenital AAT deficiency of phenotype protease inhibitor Z (PiZ) or other rare genotypes (not MS, MZ or SZ) and AAT serum level < 11 microns (µM) or < 80 mg/dL (status to be confirmed by phenotyping and genotyping)

• Inspiratory capacity (VC - ERV) > 1.2 L and forced expiratory volume at one second (FEV1) < 80% of predicted value post bronchodilator

• FEV1/VC < 70% of predicted value post-bronchodilator or transfer factor of carbon monoxide (KCO) < 80% of predicted value post-bronchodilator

• History of at least one exacerbation in the past 2 years

• Written informed consent

Exclusion Criteria:

• FEV1 < 25% of predicted value post-bronchodilator

• Augmentation therapy for more than one month with plasma-derived human alpha 1-antitrypsin (AAT) within the last 2 years

• History of lung transplant

• Any lung surgery within the past 2 years

• On any thoracic surgery waiting list

• Diagnosis of liver cirrhosis

• Severe concomitant disease

• Active pulmonary infection/exacerbations within the last month

• Active smoking during the last 6 months or plasma positive for cotinine

• Body weight < 42 kg or > 92 kg

• Pregnancy or lactation

• Women of child-bearing potential without adequate contraception