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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-08-31

Study Completion Date

2013-10-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Alpha-1 HC 100 mg

100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.

Group Type EXPERIMENTAL

Alpha-1 HC 100 mg

Intervention Type BIOLOGICAL

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.

Alpha-1 HC 200 mg

200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.

Group Type EXPERIMENTAL

Alpha-1 HC 200 mg

Intervention Type BIOLOGICAL

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.

Placebo

Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type BIOLOGICAL

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.

Interventions

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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.

Intervention Type 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.

Intervention Type 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.

Intervention Type BIOLOGICAL

Other Intervention Names

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Alpha1-proteinase inhibitor alpha1-antitrypsin Alpha1-proteinase inhibitor alpha1-antitrypsin

Eligibility Criteria

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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).

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No