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
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Condition or disease | Intervention/treatment | Phase |
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Cystic Fibrosis | Biological: Alpha-1 HC 100 mg Biological: Placebo Biological: Alpha-1 HC 200 mg | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 30 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | 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. |
Study Start Date : | August 2012 |
Actual Primary Completion Date : | October 2013 |
Actual Study Completion Date : | October 2013 |

Arm | Intervention/treatment |
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Experimental: Alpha-1 HC 100 mg
100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
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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:
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Experimental: Alpha-1 HC 200 mg
200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
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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:
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Placebo Comparator: Placebo
Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).
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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. |
- Adverse Events [ Time Frame: 3 weeks ]adverse event frequency
- 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

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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:
- 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).

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 ClinicalTrials.gov identifier (NCT number): NCT01684410
United States, Alabama | |
The University of Alabama at Birmingham | |
Birmingham, Alabama, United States, 35233 | |
United States, Colorado | |
National Jewish Hospital | |
Denver, Colorado, United States, 80206 | |
United States, Massachusetts | |
Children's Hospital Boston | |
Boston, Massachusetts, United States, 02115 | |
United States, North Carolina | |
UNC at Chapel Hill | |
Chapel Hill, North Carolina, United States, 27599 | |
United States, Ohio | |
Rainbow Babies and Children's Hospital | |
Cleveland, Ohio, United States, 44106 | |
United States, South Carolina | |
Medical University of South Carolina | |
Charleston, South Carolina, United States, 29425 |
Responsible Party: | Grifols Therapeutics LLC |
ClinicalTrials.gov Identifier: | NCT01684410 |
Other Study ID Numbers: |
T6005-201 |
First Posted: | September 13, 2012 Key Record Dates |
Results First Posted: | February 8, 2016 |
Last Update Posted: | February 8, 2016 |
Last Verified: | January 2016 |
Cystic Fibrosis Alpha1-proteinase inhibitor Alpha1-antitrypsin |
Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn |
Infant, Newborn, Diseases Protease Inhibitors Alpha 1-Antitrypsin Protein C Inhibitor Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Trypsin Inhibitors Serine Proteinase Inhibitors |