Safety and Tolerability Study of Liquid Alpha1 Proteinase Inhibitor (API) in Subjects With Cystic Fibrosis

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. Read our disclaimer for details. Identifier: NCT01347190
Recruitment Status : Completed
First Posted : May 4, 2011
Last Update Posted : April 5, 2012
Information provided by (Responsible Party):
CSL Behring

Brief Summary:
Study to assess safety and tolerability of a single dose of study-drug administered to Cystic Fibrosis (CF) patients.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Biological: CR002 Liquid API Biological: Placebo Phase 1

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Double Blind, Randomized, Placebo Controlled, Single Dose, Phase I Study of the Safety and Tolerability of Alpha1 Proteinase Inhibitor (Human) Inhalation Solution (CR002) in Subjects With Cystic Fibrosis
Study Start Date : April 2011
Actual Primary Completion Date : August 2011
Actual Study Completion Date : August 2011

Arm Intervention/treatment
Experimental: Liquid API Biological: CR002 Liquid API
A single nominal dose of CR002 (three different dose groups) will be inhaled as an aerosol mist produced by an I-neb Adaptive Aerosol Delivery (AAD) System.
Placebo Comparator: Placebo Biological: Placebo
A single nominal dose of Placebo will be inhaled as an aerosol mist produced by an I-neb Adaptive Aerosol Delivery (AAD) System.

Primary Outcome Measures :
  1. Frequency of Adverse events [ Time Frame: 44 Days (Day -21 to Day 22) ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Have a confirmed diagnosis of CF: one or more clinical findings consistent with CF and at least one of the following:

    1. Sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test OR
    2. A genotype with two identifiable mutations consistent with CF (∆F508 homozygous or two alleles known to cause a class I, II, or III mutation)
  • Have an FEV1 ≥30% of predicted normal as defined by age, gender, and height

Exclusion Criteria:

  • Oxygen saturation <90%
  • Changed in treatment regimen within 2 weeks prior to screening
  • Antibiotics regimen change < 4 weeks before screening
  • Persistent colonization with Burkholderia cepacia
  • Serum IgA < 50% of lower level of normal

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): NCT01347190

Site 11
Sofia, Bulgaria
Site 12
Varna, Bulgaria
Site 30
Debrecen, Hungary
Site 24
Bialystok, Poland
Site 22
Gdansk, Poland
Site 21
Poznan, Poland
Site 20
Rabka Zdroj, Poland
Site 23
Warszawa, Poland
United Kingdom
Site 42
Papworth Hospital, United Kingdom
Site 40
Penarth, United Kingdom
Site 41
Southampton, United Kingdom
Sponsors and Collaborators
CSL Behring
Study Director: Program director Clinical R&D CSL Behring

Responsible Party: CSL Behring Identifier: NCT01347190     History of Changes
Other Study ID Numbers: CR002_1004
2010-022671-60 ( EudraCT Number )
First Posted: May 4, 2011    Key Record Dates
Last Update Posted: April 5, 2012
Last Verified: April 2012

Keywords provided by CSL Behring:
Cystic Fibrosis

Additional relevant MeSH terms:
Cystic Fibrosis
Alpha 1-Antitrypsin Deficiency
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Liver Diseases
Subcutaneous Emphysema
Protease Inhibitors
Alpha 1-Antitrypsin
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Trypsin Inhibitors
Serine Proteinase Inhibitors