Status:
COMPLETED
Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency
Lead Sponsor:
Baxalta now part of Shire
Collaborating Sponsors:
Arriva Pharmaceuticals, Inc.
Conditions:
Alpha1-antitrypsin Deficiency
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
The study was a Phase 1B/2A, uncontrolled, open-label, single-center study in individuals with congenital AAT (alpha 1-antitrypsin) deficiency. A baseline bronchoscopy with bronchoalveolar lavage (BAL...
Eligibility Criteria
Inclusion
- Signed Informed Consent
- Male or female 18 years of age or older
- Documented, endogenous plasma AAT levels \< 11 µM (\< 80 mg/dL), either obtained from the medical history or, if not documented, plasma AAT measured after 28 day washout of any prior replacement therapy
- Forced expiratory volume at 1 second (FEV1) that is \>= 50% of predicted, measured 30 minutes after a short-acting inhaled bronchodilator
- Arterial oxygen percent saturation (SaO2, measured using room air) within the normal limits for the individual study site
- For subjects receiving an inhaled corticosteroid, inhaled or oral β-2 agonist (e.g., albuterol via metered dose inhaler \[MDI\]) or inhaled anticholinergic bronchodilator (e.g., ipratropium bromide), or oral PDE (phosphodiesterase) inhibitor, treatment on a stable dose for at least 14 days prior to enrollment
- For any female of childbearing potential, a negative urine test for pregnancy within 3 days prior to enrollment and agreement to employ adequate birth control measures for the duration of the study
- No clinically significant abnormalities detected on a 12-lead electrocardiogram (ECG) performed at the screening visit
- Laboratory results obtained at the screening visit, 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
Exclusion
- Clinically significant pulmonary impairment, other than emphysema and/or chronic bronchitis
- Moderate to severe bronchiectasis
- 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 within 28 days prior to the screening visit
- 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
- Prior history of adverse reactions to the local anesthetic, sedative, BAL procedure, or pre-medication employed at the study center
- Use of oral or parenteral glucocorticosteroids, or alpha 1-antitrypsin replacement therapy within 28 days prior to baseline BAL, or any use planned during the study. However, the subject may enroll provided that a) consent is given to undergo a 28-day washout of the replacement or steroid therapy, and b) no study procedures are done until the washout is completed.
- Use of another investigational drug or investigational device within 28 days prior to baseline BAL
- Any upper or lower respiratory infection within 28 days prior to baseline BAL
- Having received a lung or liver transplant
Key Trial Info
Start Date :
March 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 14 2004
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT00157092
Start Date
March 1 2004
End Date
October 14 2004
Last Update
May 5 2021
Active Locations (1)
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1
Shands Hospital at the University of Florida
Gainesville, Florida, United States, 32610