Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency
Phase 1
Completed
- Conditions
- Alpha1-antitrypsin Deficiency
- Registration Number
- NCT00161707
- Lead Sponsor
- Baxalta now part of Shire
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 40
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Primary Outcome Measures
Name Time Method Number of participants who develop antibodies to Recombinant Alpha 1-Antitrypsin (rAAT) 6 weeks after the first inhalation of study drug
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (4)
Shands Hospital at the University of Florida
🇺🇸Gainesville, Florida, United States
National Jewish Medical and Research Center
🇺🇸Denver, Colorado, 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