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Trial record 7 of 10 for:    Alpha-1-proteinase inhibitor AND ELANE


The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02525861
Recruitment Status : Recruiting
First Posted : August 18, 2015
Last Update Posted : February 15, 2019
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Brief Summary:
The purpose of the study is 2-fold: (1) to further evaluate the safety and potential immunogenicity of GLASSIA following IV administration via in-line filtration; and, (2) to assess the effects of GLASSIA augmentation therapy on the levels of A1PI and various biomarkers in the epithelial lining fluid (ELF) following intravenous (IV) administration at a dosage of 60 mg/kg Body weight (BW)/week active A1PI protein for 25 weeks in subjects with emphysema due to congenital A1PI deficiency.

Condition or disease Intervention/treatment Phase
Alpha1-antitrypsin Deficiency Biological: GLASSIA Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Actual Study Start Date : April 21, 2016
Estimated Primary Completion Date : May 29, 2020
Estimated Study Completion Date : May 29, 2020

Arm Intervention/treatment
Active Comparator: GLASSIA with lower particulate level
GLASSIA lot(s) produced within acceptable range, with lower particulate level
Biological: GLASSIA
60 mg/kg BW administered at a rate of 0.2 mL/kg/min

Active Comparator: GLASSIA with higher particulate level
GLASSIA lot(s) produced within acceptable range, with higher particulate level
Biological: GLASSIA
60 mg/kg BW administered at a rate of 0.2 mL/kg/min

Primary Outcome Measures :
  1. Number (proportion) of adverse events (AEs) considered potentially related to the presence of particle load in the GLASSIA solution [ Time Frame: 6 months ]
  2. Incidence of treatment-emergent adverse reactions (ARs) plus suspected ARs [ Time Frame: 6 months ]
  3. Number (proportion) of infusions that are discontinued, slowed, or interrupted due to an adverse event (AE) [ Time Frame: 6 months ]
  4. Number (proportion) of participants who develop binding and/or neutralizing anti-A1PI antibodies [ Time Frame: 6 months ]
  5. Antigenic A1PI levels in ELF [ Time Frame: 12 - 14 weeks ]
  6. Functional A1PI (also known as anti-neutrophil elastase capacity [ANEC]) levels in ELF [ Time Frame: 12 - 14 weeks ]

Secondary Outcome Measures :
  1. Incidence of treatment-emergent adverse events (AEs) [ Time Frame: 6 months ]
  2. Number (proportion) of participants who experienced a shift from normal or clinically insignificant abnormal laboratory values at baseline to clinically significant abnormal laboratory values following GLASSIA administration [ Time Frame: 6 months ]
  3. Number (proportion) of subjects with treatment-emergent seroconversion or positive nucleic acid test (NAT) for parvovirus B19 (B19V) [ Time Frame: 6 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male or female participants meeting the following age criteria:

    1. For participants who will undergo bronchoscopy/ bronchoalveolar lavage (BAL) procedures: 18 to 75 years of age at the time of screening.
    2. For participants who will be waived from undergoing bronchoscopy/BAL procedures: 18 years of age or older at the time of screening.
  2. Documented Alpha1-Proteinase Inhibitor (A1PI) genotype of Pi*Z/Z, Pi*Z/Null, Pi*Malton/Z, Pi*Null/Null, or other "at-risk" allelic combinations such as SZ (excluding MS and MZ) and an endogenous A1PI plasma levels of ≤11 μM.
  3. Screening levels of endogenous plasma (antigenic) A1PI of ≤11 μM may be collected at any time during the screening period for treatment-naïve participants, or following a 4 week minimum wash-out from previous augmentation therapy in treatment-experienced participants.
  4. Participants must have at least one of the following: clinical diagnosis of emphysema, evidence of emphysema on computerized tomography (CT) scan of the chest, and/or evidence of airway obstruction which is not completely reversed with bronchodilator treatment at the time of screening.
  5. If the participant is being treated with any respiratory medications including inhaled bronchodilators, inhaled anticholinergics, inhaled corticosteroids, or low-dose systemic corticosteroids (prednisone ≤10 mg/day or its equivalent), the doses of the participant's medications have remained unchanged for at least 14 days prior to screening.
  6. The participant is a nonsmoker or has ceased smoking for a minimum of 13 weeks prior to screening (serum cotinine level at screening within normal range of a nonsmoker) and agrees to refrain from smoking throughout the course of the study. Participants with a positive cotinine test due to nicotine replacement therapy (eg, patches, chewing gum), vapor cigarettes, or snuff are eligible.
  7. If female of childbearing potential, the participant presents with a negative pregnancy test at screening and agrees to employ adequate birth control measures for the duration of the study.
  8. The participant is willing and able to comply with the requirements of the protocol.
  9. The participant must have pulmonary function at the time of screening meeting both of the following:

    1. Post-bronchodilator forced expiratory volume in 1 second (FEV1) ≥50% of predicted.
    2. If FEV1 is >80% predicted, then FEV1/forced vital capacity (FVC) must be <0.7. *Note: Inclusion criterion #1a and #9a are not applicable to participants who are not required to undergo the bronchoscopy/BAL procedures.

Exclusion Criteria:

  1. The participant is experiencing or has a history of clinically significant pulmonary disease (other than chronic obstructive pulmonary disease (COPD), emphysema, chronic bronchitis, mild bronchiectasis, and stable asthma).
  2. The participant is experiencing or has a history of chronic severe cor pulmonale (resting mean pulmonary artery pressure ≥40 millimeter(s) of mercury (mm Hg)).
  3. The participant routinely produces more than 1 tablespoon of sputum per day.
  4. The participant has a history of frequent pulmonary exacerbations (greater than 2 moderate or severe exacerbations within 52 weeks prior to screening.
  5. The participant is experiencing a pulmonary exacerbation at the time of screening (participant may be re-screened 4 weeks after the clinical resolution of an exacerbation).
  6. The participant has clinically significant abnormalities (other than emphysema, chronic bronchitis, or mild bronchiectasis) detected on chest X-ray or CT scan at the time of screening. (Past records obtained within 52 weeks prior to screening may be used, if available.)
  7. The participant has clinically significant abnormalities detected on a 12-lead electrocardiogram (ECG) performed at the time of screening. (Past records obtained within 26 weeks prior to screening may be used, if available.)
  8. The participant has clinically significant congestive heart failure with New York Heart Association (NYHA) Class III/IV symptoms.
  9. The participant is experiencing an active malignancy or has a history of malignancy within 5 years prior to screening, with the exception of the following: adequately treated basal cell or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, or stable prostate cancer not requiring treatment.
  10. The participant has a history of lung or other organ transplant, is currently on a transplant list, or has undergone major lung surgery.
  11. The participant is receiving long-term around-the-clock oxygen supplementation. (The following are allowed: short-term use of oxygen supplementation [eg, for the management of acute COPD exacerbation], oxygen supplementation required during night time only, and supplemental oxygen (O2) with continuous positive airway pressure [CPAP] or bi-level positive airway pressure [BiPAP]).
  12. Known history of hypersensitivity following infusions of human blood or blood components.
  13. Immunoglobulin A (IgA) deficiency (<8 mg/dL at screening).
  14. Abnormal clinical laboratory results obtained at the time of screening meeting any of the following criteria:

    1. Serum alanine aminotransferase (ALT) >3.0 times upper limit of normal (ULN)
    2. Serum total bilirubin >2.0 times ULN
    3. >2+proteinuria on urine dipstick analysis
    4. Serum creatinine >2.0 times ULN
    5. Absolute neutrophil count (ANC) <1500 cells/mm^3
    6. Hemoglobin (Hgb) <9.0 g/dL
    7. Platelet count <100,000/mm^3
  15. Ongoing active infection with hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) Type 1 or 2 infection at the time of screening.
  16. The participant has any clinically significant medical, psychiatric, or cognitive illness, or any other uncontrolled medical condition (eg, unstable angina, transient ischemic attack) that, in the opinion of the investigator, would impede the participant's ability to comply with the study procedures, pose increased risk to the participant's safety, or confound the interpretation of study results.
  17. The participant has participated in another clinical study involving an investigational product or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an investigational product or device during the course of this study.
  18. The participant is a family member or employee of the investigator.
  19. If female, the participant is nursing at the time of screening.
  20. The participant has contraindication(s) to bronchoscopy such as recent myocardial infarction, unstable angina, other cardiopulmonary instability, tracheal obstruction or stenosis, moderate to severe hypoxemia or any degree of hypercapnia, unstable asthma, Stage 4 or 5 chronic kidney disease, pulmonary hypertension, severe hemorrhagic diathesis, and cervical C1/C2 arthritis.
  21. The participant has had lung surgery which may interfere with bronchoscopy.
  22. Known history of allergic/hypersensitivity reactions to medications used during and for perioperative care associated with the bronchoscopy/BAL procedures, such as local anesthetics, sedatives, pain control medications.
  23. The participant is receiving or requires long-term (>4 weeks) immunosuppressive therapy, such as systemic corticosteroids at doses greater than 10 mg/day of prednisone (or its equivalent), mycophenolate mofetil, azathioprine, cyclophosphamide, and rituximab.
  24. If a participant is receiving anticoagulant or anti-platelet therapy (such as warfarin and clopidogrel), the participant is unwilling to or unable to safely discontinue anticoagulant or anti-platelet therapy within 7 days prior to until at least 24 hours after the BAL procedures. An exception is low-dose aspirin alone which is allowed.

    • Note: Exclusion criteria #20, #21, #22, #23, and #24 are not applicable to participants who are not required to undergo the bronchoscopy/BAL procedures.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02525861

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Contact: Shire Contact +1 866 842 5335

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United States, Arizona
St. Joseph's Hospital and Medical Center Withdrawn
Phoenix, Arizona, United States, 85013
Arizona Board of Regents, University of Arizona Recruiting
Tucson, Arizona, United States, 85724
Contact: Site Contact    844-242-4664   
Principal Investigator: Tony Hodges, MD         
United States, California
UCLA Medical Center Recruiting
Los Angeles, California, United States, 90024
Contact: Site Contact    310-206-0396   
Principal Investigator: Igor Barjaktarevic, MD         
Cedars Sinai Medical Center, Division of Pulmonary and Critical Care Medicine Recruiting
Los Angeles, California, United States, 90048
Contact: Site Contact    310-423-4765   
Principal Investigator: Jeremy Falk, MD         
University of California Davis Health System Recruiting
Sacramento, California, United States, 95817
Contact: Site Contact    916-703-4093   
Principal Investigator: Brian Morrissey, MD         
United States, Florida
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact: Site Contact    305-243-2568   
Principal Investigator: Michael Campos, MD         
DBC Research Corp, Pembroke Pines Recruiting
Tamarac, Florida, United States, 33321
Contact: Site Contact    954-537-2100   
Principal Investigator: Alvaro Murcia, MD         
Tampa General Hospital Withdrawn
Tampa, Florida, United States, 33606
Cleveland Clinic Florida - Weston Recruiting
Weston, Florida, United States, 33331
Contact: Site Contact    954-659-6409   
Principal Investigator: Franck Rahaghi, MD         
United States, Illinois
University of Chicago Medical Center Recruiting
Chicago, Illinois, United States, 60637
Contact: Site Contact    773-702-1012   
Principal Investigator: Douglas Hogarth, MD         
United States, Kentucky
Kentucky Lung Clinic, PSC Withdrawn
Hazard, Kentucky, United States, 41701
United States, Maryland
Pulmonary Critical Care Associates of Baltimore Recruiting
Towson, Maryland, United States, 21286
Contact: Site Contact    410-494-1662   
Principal Investigator: Linda Barr, MD         
United States, Missouri
Hannibal Clinic Recruiting
Hannibal, Missouri, United States, 63401
Contact: Site Contact    573-231-3239   
Principal Investigator: Humam Farah, MD         
United States, North Carolina
Southeastern Research Center LLC Recruiting
Winston-Salem, North Carolina, United States, 27103
Contact: Site Contact    336-659-8414   
Principal Investigator: Barry Sigal, MD         
United States, Pennsylvania
Temple University School of Medicine Recruiting
Philadelphia, Pennsylvania, United States, 19140
Contact: Site Contact    215-707-4679   
Principal Investigator: Friedrich Kueppers, MD         
United States, South Carolina
Medical University of South Carolina (MUSC) Recruiting
Charleston, South Carolina, United States, 29425
Contact: Site Contact    843-792-8438   
Principal Investigator: Charlton Strange, MD         
United States, Tennessee
Clinical Research Solutions, LLC Withdrawn
Franklin, Tennessee, United States, 37067
United States, Texas
Renovatio Clinical-Respiratory & Sleep Disorders Specialists Recruiting
The Woodlands, Texas, United States, 77005
Contact: Site Contact    713-703-2398   
Principal Investigator: Ather Siddiqi, MD         
University of Texas Health Science Center at Tyler Recruiting
Tyler, Texas, United States, 75708
Contact: Site Contact    903-877-5518   
Principal Investigator: James Stocks, MD         
Canada, Ontario
LHSC - Victoria Hospital Recruiting
London, Ontario, Canada, N6A 5W9
Contact: Site Contact    519-685-8500 ext 76094   
Principal Investigator: David McCormack, MD         
Sponsors and Collaborators
Baxalta now part of Shire
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Study Director: Study Director Shire

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Responsible Party: Baxalta now part of Shire Identifier: NCT02525861     History of Changes
Other Study ID Numbers: 471101
First Posted: August 18, 2015    Key Record Dates
Last Update Posted: February 15, 2019
Last Verified: February 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency
Alpha 1-Antitrypsin
Trypsin Inhibitors
Serine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Pathologic Processes
Molecular Mechanisms of Pharmacological Action