Status:
COMPLETED
Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)
Lead Sponsor:
Grifols Therapeutics LLC
Conditions:
Alpha 1-Antitrypsin Deficiency
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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 alph...
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 densit...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
December 1 2003
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2007
Estimated Enrollment :
77 Patients enrolled
Trial Details
Trial ID
NCT00263887
Start Date
December 1 2003
End Date
January 1 2007
Last Update
August 21 2014
Active Locations (3)
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1
Gentofte Hospital Department of Respiratory Medicine
Hellerup, Denmark, 2900
2
Department of Pulmonary Medicine, Malmö University Hospital
Malmo, Sweden
3
Queen Elizabeth Hospital
Birmingham, England, United Kingdom, B15 2TH