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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01347190
First received: May 3, 2011
Last updated: April 3, 2012
Last verified: April 2012
  Purpose

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


Condition Intervention Phase
Cystic Fibrosis
Biological: CR002 Liquid API
Biological: Placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Frequency of Adverse events [ Time Frame: 44 Days (Day -21 to Day 22) ] [ Designated as safety issue: Yes ]

Enrollment: 25
Study Start Date: April 2011
Study Completion Date: August 2011
Primary Completion Date: August 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01347190

Locations
Bulgaria
Site 11
Sofia, Bulgaria
Site 12
Varna, Bulgaria
Hungary
Site 30
Debrecen, Hungary
Poland
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
Investigators
Study Director: Program director Clinical R&D CSL Behring
  More Information

No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01347190     History of Changes
Other Study ID Numbers: CR002_1004, 2010-022671-60
Study First Received: May 3, 2011
Last Updated: April 3, 2012
Health Authority: Bulgaria: Ministry of Health
Hungary: National Institute of Pharmacy
Poland: Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by CSL Behring:
Cystic Fibrosis

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

ClinicalTrials.gov processed this record on September 16, 2014