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

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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

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.
    


Study Phase

Phase 1/Phase 2

Study Type

Interventional




Condition

Alpha1-antitrypsin Deficiency

Intervention

Aerosolized, Recombinant Alpha 1-Antitrypsin


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug


Start Date

January 1, 2003

Completion Date

October 1, 2003


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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Study Director, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00161707

Organization ID

410103


Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Collaborators

 Arriva Pharmaceuticals, Inc.

Study Sponsor

Study Director, Study Director, Shire


Verification Date

July 2019