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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00263887
• Date of registration: 12/09/2005
• Prospective Registration: No
• Primary sponsor: Grifols Therapeutics Inc.
• Public title: Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)
• Scientific title: 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.
• Date of first enrolment: December 2003
• Target sample size: 77
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00263887
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Denmark, Sweden, United Kingdom

Contacts

• Name: Asger Dirksen, MD PHD
• Affiliation: University of Copenhagen

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Alpha 1-Antitrypsin Deficiency

Intervention(s)

Drug: Albumin (Human) 20%, United States Pharmacopeia (USP)

Drug: Alpha1-Proteinase Inhibitor (Human)

Primary Outcome(s)

The Progression Rate of Emphysema Determined by Change in 15th Percentile of Lung Density Measured by Annual CT Scan of the Whole Lung [Time Frame: 24 or 30 months]

Secondary Outcome(s)

Mortality [Time Frame: 24 or 30 months]

The Frequency of Exacerbations as Determined by Patient Diary. [Time Frame: 24 or 30 months]

Change in Lung Density at Each Visit as Measured by Computed Tomography [Time Frame: 24 or 30 months]

Duration and Severity of the Exacerbations [Time Frame: 24 or 30 months]

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) [Time Frame: 24 or 30 months]

Quality of Life With a Disease Specific Instrument, the St. George's Respiratory Questionnaire [Time Frame: 24 or 30 months]

Secondary ID(s)

100533

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 21/08/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00263887