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Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT01684410
Recruitment Status : Completed
First Posted : September 13, 2012
Results First Posted : February 8, 2016
Last Update Posted : February 8, 2016
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics LLC

Tracking Information
First Submitted Date  ICMJE September 6, 2012
First Posted Date  ICMJE September 13, 2012
Results First Submitted Date  ICMJE August 10, 2015
Results First Posted Date  ICMJE February 8, 2016
Last Update Posted Date February 8, 2016
Study Start Date  ICMJE August 2012
Actual Primary Completion Date October 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 11, 2016)
Adverse Events [ Time Frame: 3 weeks ]
adverse event frequency
Original Primary Outcome Measures  ICMJE
 (submitted: September 10, 2012)
  • Acute pulmonary exacerbations [ Time Frame: 3 weeks ]
    The number of subjects with signs and symptoms of acute pulmonary exacerbation, by severity and duration.
  • Adverse Events [ Time Frame: 3 weeks ]
    The number of subjects with adverse events.
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: January 11, 2016)
  • Percent Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Week 3 [ Time Frame: 3 weeks ]
    FEV1 conducted before and after inhalation of the investigational product at study visits.
  • Percent Change From Baseline in Forced Vital Capacity (FVC) at Week 3 [ Time Frame: 3 weeks ]
    FVC conducted before and after inhalation of the investigational product
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis
Official Title  ICMJE A Three Week Dose Escalation, Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Safety and Tolerability of 100 mg or 200 mg of Inhaled Alpha-1 HC, Once a Day in Subjects With Cystic Fibrosis.
Brief Summary This was a randomized, double-blind, placebo-controlled, dose escalation study to assess the safety and tolerability of 100 mg and 200 mg of inhaled Alpha-1 HC administered once a day for three weeks in subjects aged 18 years and older with cystic fibrosis (CF). The treatment duration in this study was intended to provide multi-dose safety information prior to proceeding to longer durations of exposure.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Biological: Alpha-1 HC 100 mg
    Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 100 mg inhaled once daily for 21 days for a total of 21 inhaled treatments.
    Other Names:
    • Alpha1-proteinase inhibitor
    • alpha1-antitrypsin
  • Biological: Placebo
    Phosphate Buffer Saline with Polysorbate (placebo) composed of the same elements listed for Alpha-1 HC, minus the 50 mg/mL of Alpha-1 HC. Placebo inhaled once daily for 21 days for a total of 21 inhaled treatments.
  • Biological: Alpha-1 HC 200 mg
    Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 200 mg inhaled once daily for 21 days for a total of 21 inhaled treatments.
    Other Names:
    • Alpha1-proteinase inhibitor
    • alpha1-antitrypsin
Study Arms  ICMJE
  • Experimental: Alpha-1 HC 100 mg
    100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
    Intervention: Biological: Alpha-1 HC 100 mg
  • Experimental: Alpha-1 HC 200 mg
    200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
    Intervention: Biological: Alpha-1 HC 200 mg
  • Placebo Comparator: Placebo
    Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).
    Intervention: Biological: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 10, 2012)
30
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE October 2013
Actual Primary Completion Date October 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Age 18 years or older.
  • Documentation of CF diagnosis.
  • Have a pre-bronchodilator FEV1 ≥ 40% of predicted at Visit 1 and have a Visit 2 pre-investigational product FEV1 that is ≥ 40% of predicted and within ± 15% of the Visit 1 result.
  • Deemed by the Investigator to be a suitable candidate for serial collection of expectorated sputum.

Exclusion Criteria:

  • Had a pulmonary exacerbation during the 4 weeks before screening (Visit 1) which required the initiation of new antibiotic treatment
  • Have a pulmonary exacerbation during the screening period (between Visit 1 and Visit 2) which requires the initiation of new antibiotic treatment
  • FEV1 < 0.59 liters at the screening visit
  • Respiratory insufficiency with continuous supplemental oxygen therapy, or carbon dioxide retention
  • Elevated aspartate transaminase (AST) or alanine aminotransferase (ALT) that is ≥ 3 times the upper limit of normal for age and gender
  • Smoking during the past 6 months
  • Lung surgery during the past 2 years
  • Positive culture for Burkholderia cepacia or mycobacterium during the past two years.
  • Active allergic bronchopulmonary aspergillosis
  • Pre-treatment sputum collection at Visit 1 or Visit 2 (Randomization) characterized by problems such as inadequate sputum volume or quality.
  • Known selective Immunoglobulin A (IgA) deficiency with known antibody against IgA (anti-IgA antibody).
  • History of anaphylaxis or severe systemic response to any plasma-derived alpha1-proteinase inhibitor preparation or other blood product(s), or to polysorbates.
  • Use of chronic oral steroids during the study. Note: Inhaled corticosteroids that had been administered for at least 4 weeks prior to Visit 1 were permissible during the study.
  • Use of chronic, high dose ibuprofen therapy within 3 weeks of screening and at anytime during the study.
  • Chronic maintenance therapy with systemic antibiotics within 3 weeks of screening and through last dose of investigational product.
  • Use of leukotriene synthesis inhibitor (zileuton) or leukotriene receptor antagonists (montelukast, zafirlukast) within 3 weeks of screening and at anytime during the study.
  • Use of roflumilast within 3 weeks of screening and at any time during the study.
  • Initiation of a new chronic medication or dosage change of a chronic medication for treatment of cystic fibrosis (example: Kalydeco™ [ivacaftor]) within 3 weeks of screening (Visit 1).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01684410
Other Study ID Numbers  ICMJE T6005-201
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Grifols Therapeutics LLC
Study Sponsor  ICMJE Grifols Therapeutics LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Grifols Therapeutics LLC
Verification Date January 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP