NCT00263887

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

Multi-center, Randomized Trial With I.V. Prolastin® to Evaluate Frequency of Exacerbations and Progression of Emphysema by Means of Multi-slice CT Scans in Patients With Congenital Alpha-1-antitrypsin Deficiency.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 77 (actual)
• Sponsor: Grifols Therapeutics LLC · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a one to one randomized, placebo-controlled, clinical, exploratory study with the aim of collecting information on possible clinical endpoints i.e., the progression of emphysema by lung density measurements with CT scan and frequency of exacerbations that could be used for a subsequent placebo controlled clinical trial. Progression of disease will be investigated in 80 patients with alpha-1-antitrypsin deficiency, who will be treated with human alpha-1-antitrypsin (AAT; Prolastin®) or placebo weekly for two years to analyze the effect of treatment on lung density and exacerbations. Targeted augmentation therapy with weekly infusions of Prolastin® will be a dose of 60 mg/kg body weight (range of 51.72 to 71.43 mg per kg body weight). Therefore, this study focuses on several questions: * Is the 15th percentile point calculated by analysis of CT lung histograms a useful endpoint for clinical trials in AAT deficiency? * Is quantitation of exacerbations in AAT-deficient patients a useful endpoint for clinical trials in AAT deficiency? * Are there significant differences between the treatments in favor of Prolastin®?

Conditions

• Alpha 1-Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	Alpha1-Proteinase Inhibitor (Human)	Weekly infusion of 60 mg/kg body weight for 2 years
DRUG	Albumin (Human) 20%, United States Pharmacopeia (USP)	Weekly infusion for 2 years. Albumin (Human) 20% will be diluted with 5% glucose to a final concentration of 2.0%.

Timeline

• Start date: 2003-12-01
• Primary completion: 2007-01-01
• Completion: 2007-01-01
• First posted: 2005-12-09
• Last updated: 2014-08-21
• Results posted: 2014-08-21

Locations

3 sites across 3 countries: Denmark, Sweden, United Kingdom