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

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Baxalta

Status and phase

Completed
Phase 2
Phase 1

Conditions

Alpha1-antitrypsin Deficiency

Treatments

Drug: Aerosolized, Recombinant Alpha 1-Antitrypsin

Study type

Interventional

Funder types

Industry

Identifiers

Details and patient eligibility

About

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.

Enrollment

40 patients

Sex

All

Ages

18+ years old

Volunteers

No 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

Trial contacts and locations

4

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Data sourced from clinicaltrials.gov

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