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

Primary Purpose

Alpha1-antitrypsin Deficiency

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Aerosolized, Recombinant Alpha 1-Antitrypsin
Sponsored by
Baxalta now part of Shire
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Alpha1-antitrypsin Deficiency

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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 < 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

Sites / Locations

  • National Jewish Medical and Research Center
  • Shands Hospital at the University of Florida
  • Cleveland Clinic Foundation, Department of Pulmonary and Critical Care Medicine
  • The University of Texas Health Science Center at Tyler

Outcomes

Primary Outcome Measures

Number of participants who develop antibodies to Recombinant Alpha 1-Antitrypsin (rAAT)

Secondary Outcome Measures

Full Information

First Posted
September 8, 2005
Last Updated
April 29, 2021
Sponsor
Baxalta now part of Shire
Collaborators
Arriva Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT00161707
Brief Title
Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency
Official Title
Phase I Safety Investigation of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency
Study Type
Interventional

2. Study Status

Record Verification Date
April 2021
Overall Recruitment Status
Completed
Study Start Date
January 7, 2003 (Actual)
Primary Completion Date
October 1, 2003 (Actual)
Study Completion Date
October 1, 2003 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Baxalta now part of Shire
Collaborators
Arriva Pharmaceuticals, Inc.

4. Oversight

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alpha1-antitrypsin Deficiency

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Crossover Assignment
Allocation
Randomized
Enrollment
40 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Aerosolized, Recombinant Alpha 1-Antitrypsin
Primary Outcome Measure Information:
Title
Number of participants who develop antibodies to Recombinant Alpha 1-Antitrypsin (rAAT)
Time Frame
6 weeks after the first inhalation of study drug

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
National Jewish Medical and Research Center
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Facility Name
Shands Hospital at the University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Cleveland Clinic Foundation, Department of Pulmonary and Critical Care Medicine
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
The University of Texas Health Science Center at Tyler
City
Tyler
State/Province
Texas
ZIP/Postal Code
75708-3154
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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