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Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00161707
Recruitment Status : Completed
First Posted : September 13, 2005
Last Update Posted : October 23, 2017
Sponsor:
Collaborator:
Information provided by:

Study Description
Brief Summary:
The purpose of this randomized, double-blind, placebo-controlled study is to evaluate the short-term safety of inhaled recombinant alpha 1-antitrypsin (rAAT) in subjects with alpha 1-antitrypsin deficiency. The subjects are randomized to receive placebo or one of 4 doses of rAAT. The 4 doses are tested in a consecutive manner from lowest to highest.

Condition or disease Intervention/treatment Phase
Alpha 1-antitrypsin Deficiency Drug: Aerosolized, Recombinant Alpha 1-Antitrypsin Phase 1 Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: Phase I Safety Investigation of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency
Study Start Date : January 2003
Estimated Study Completion Date : October 2003


Arms and Interventions


Outcome Measures

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female 18 years of age or older
  • Endogenous plasma AAT levels < 11 µM (< 80 mg/dL)
  • Baseline forced expiratory volume at one second (FEV1) that is >= 50% of predicted, measured 30 minutes after a short-acting inhaled bronchodilator
  • Baseline arterial oxygen percent saturation (SaO2) within the normal limits for the individual study site
  • For subjects receiving an inhaled corticosteroid, β-2 agonist (eg, albuterol via metered dose inhaler [MDI]) or anticholinergic bronchodilator (eg, ipratropium bromide), treatment on a stable dose for at least 14 days prior to randomization
  • If female of childbearing potential, negative urine pregnancy test within 3 days prior to randomization and agreement to employ adequate birth control measures
  • No clinically significant abnormalities detected on a 12-lead electrocardiogram (ECG) performed no more than 7 days prior to randomization
  • Baseline laboratory results, obtained no more than 7 days prior to randomization, meeting the following criteria:
  • Serum aspartate transaminase (AST) and alanine transaminase (ALT) <= 2 times upper limit of normal range (ULN)
  • Serum total bilirubin <= 2 times ULN
  • < 2+ proteinuria on urine dipstick
  • Serum creatinine <= 1.5 times ULN
  • Absolute neutrophil count >= 1500 cells/mm3
  • Hemoglobin >= 10.0 g/dL
  • Platelet count >= 100,000/mm3
  • Signed informed consent

Exclusion Criteria:

  • Clinically significant pulmonary impairment, other than emphysema and/or chronic bronchitis
  • Clinically significant cardiac, hemostatic, or neurologic impairment, or other significant medical condition that, in the opinion of the investigator, would affect subject safety or compliance
  • Psychiatric or cognitive disturbance or illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance
  • Acute exacerbation of emphysema (as defined in Section 8.5.10) within 28 days prior to randomization
  • Pregnancy or lactation
  • Known history of allergy to yeast products
  • Medical history precluding the use of epinephrine or other rescue medication for treatment of anaphylaxis
  • Use of antihistamines within 7 days prior to randomization
  • Use of oral steroids, beta-blockers, or tricyclic antidepressants within 28 days prior to randomization
  • Use of another investigational drug or investigational device within 28 days prior to randomization
  • Any upper or lower respiratory infection within 28 days prior to randomization
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00161707


Locations
United States, Colorado
National Jewish Medical and Research Center
Denver, Colorado, United States, 80206
United States, Florida
Shands Hospital at the University of Florida
Gainesville, Florida, United States, 32610
United States, Ohio
Cleveland Clinic Foundation, Department of Pulmonary and Critical Care Medicine
Cleveland, Ohio, United States, 44195
United States, Texas
The University of Texas Health Science Center at Tyler
Tyler, Texas, United States, 75708-3154
Sponsors and Collaborators
Baxalta now part of Shire
Arriva Pharmaceuticals, Inc.
Investigators
Principal Investigator: Mark Brantly, MD Shands Hospital at the University of Florida
More Information

ClinicalTrials.gov Identifier: NCT00161707     History of Changes
Other Study ID Numbers: 410103
First Posted: September 13, 2005    Key Record Dates
Last Update Posted: October 23, 2017
Last Verified: October 2006

Additional relevant MeSH terms:
Alpha 1-Antitrypsin Deficiency
Alpha 1-Antitrypsin
Protein C Inhibitor
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes
Trypsin Inhibitors
Serine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action