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

Phase I Safety Investigation of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency

NCT00161707•Baxalta now part of Shire

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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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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
+6 more criteria

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
+1 more criteria

Locations (4)

National Jewish Medical and Research Center

Denver, Colorado, United States

Shands Hospital at the University of Florida

Gainesville, Florida, United States

Cleveland Clinic Foundation, Department of Pulmonary and Critical Care Medicine

Cleveland, Ohio, United States

The University of Texas Health Science Center at Tyler

Tyler, Texas, United States

Key Dates

Start Date

January 7, 2003

Primary Completion Date

October 1, 2003

Completion Date

October 1, 2003

Last Updated

May 5, 2021

Enrollment

ACTUAL participants

Conditions

Alpha1-antitrypsin Deficiency

Interventions

Aerosolized, Recombinant Alpha 1-Antitrypsin

DRUG

A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency

NCT04722887

Alpha1-Antitrypsin Deficiency

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: May 5, 2021Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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