Phase 2 Completed N=77 Randomized Quadruple-blind Treatment

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

Source: ClinicalTrials.gov NCT00263887 ↗

Enrolled (actual)

Serious AEs

31.2%

Results posted

Aug 2014

Primary outcomePrimary: The Progression Rate of Emphysema Determined by Change in 15th Percentile of Lung Density Measured by Annual CT Scan of the Whole Lung — 47.980; 45.477; 45.085; 41.354 g/L — p=0.049

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.

Outcome Measures

Outcome	Result	p-value
PRIMARY The Progression Rate of Emphysema Determined by Change in 15th Percentile of Lung Density Measured by Annual CT Scan of the Whole Lung	47.980; 45.477; 45.085; 41.354; -2.895; -4.124	0.049 sig
SECONDARY Change in Lung Density at Each Visit as Measured by Computed Tomography	—	—
SECONDARY The Frequency of Exacerbations as Determined by Patient Diary.	—	—
SECONDARY The Deterioration of the Lung Function Will be Assessed by Measurement of the Change in Forced Expiratory Volume at One Second (FEV1) and Transfer Factor of Carbon Monoxide (KCO)	—	—
SECONDARY Duration and Severity of the Exacerbations	—	—
SECONDARY Mortality	—	—
SECONDARY Quality of Life With a Disease Specific Instrument, the St. George's Respiratory Questionnaire	—	—

Eligibility 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 1.2 L and forced expiratory volume at one second (FEV1) 92 kg
Pregnancy or lactation
Women of child-bearing potential without adequate contraception

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Data sourced from ClinicalTrials.gov (NCT00263887). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.