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Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.

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ClinicalTrials.gov Identifier: NCT00670007
Recruitment Status : Completed
First Posted : May 1, 2008
Results First Posted : July 12, 2016
Last Update Posted : August 15, 2016
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Tracking Information
First Submitted Date  ICMJE April 29, 2008
First Posted Date  ICMJE May 1, 2008
Results First Submitted Date  ICMJE June 2, 2016
Results First Posted Date  ICMJE July 12, 2016
Last Update Posted Date August 15, 2016
Study Start Date  ICMJE April 2008
Actual Primary Completion Date September 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 2, 2016)
Rate of Change of Adjusted Lung Density [ Time Frame: Up to 2 years ]
As measured by centralized, standardized computer tomographic (CT) lung densitometry. CT scans were acquired at 2 inspiration states: TLC (Total Lung Capacity; ie, full inspiration) and FRC (Functional Residual Capacity; ie, full expiration). Results were adjusted for total lung volume and are presented as point estimates for the average rate of decline in the early start and delayed start subgroups from a linear random regression model with country, inspiration state (only for 'TLC and FRC state'), time (time elapsed since Day 1 [CE1226_4001]), treatment and treatment by time interaction as fixed effects and subject and subject by time interaction as random coefficients.
Original Primary Outcome Measures  ICMJE
 (submitted: April 30, 2008)
Lung Density measured by CT scans [ Time Frame: Yearly ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 13, 2016)
  • Absolute Change in Adjusted Lung Density [ Time Frame: From baseline to 2 years ]
    Absolute change from baseline to 2 years as measured by centralized, standardized CT lung densitometry. CT scans were acquired at 2 inspiration states: TLC (ie, full inspiration) and FRC (ie, full expiration). Results were adjusted for total lung volume and are presented as point estimates for the average absolute change in the early start and delayed start subgroups from an analysis of covariance (ANCOVA) model with country, treatment, and baseline lung density as fixed effects and inspiration state as a repeated random effect. The baseline is the last assessment from the preceding study CE1226_4001.
  • Percent Change in Adjusted Lung Density [ Time Frame: From baseline to 2 years ]
    Percent change from baseline to 2 years as measured by centralized, standardized CT lung densitometry. CT scans were acquired at 2 inspiration states: TLC (ie, full inspiration) and FRC (ie, full expiration). Results were adjusted for total lung volume and are presented as point estimates for the average percent change in the early start and delayed start subgroups from an analysis of covariance (ANCOVA) model with country, treatment, and baseline lung density as fixed effects and inspiration state as a repeated random effect. The baseline is the last assessment from the preceding study CE1226_4001.
  • Change in Subject-reported Symptoms [ Time Frame: From baseline to 2 years ]
    Patient-reported symptoms were measured using the St George's Respiratory Questionnaire (SGRQ). SGRQ total, symptoms, activity and impact scores range from 0 to 100, with higher scores indicating more limitations, and change from baseline below zero (0) is favorable, indicating improvement.
  • Percent Change in Lung Function as Measured by Forced Expiratory Volume in 1 Second (FEV1) [ Time Frame: From baseline up to 2 years ]
  • Percent Change in Lung Function as Measured by Ratio of FEV1/FVC (Forced Vital Capacity) [ Time Frame: From baseline up to 2 years ]
  • Percent Change in Lung Function as Measured by Percent Predicted FEV1 [ Time Frame: From baseline up to 2 years ]
  • Number of Subjects With Pulmonary Exacerbations [ Time Frame: Up to 2 years ]
  • Annual Rate in Subject Years of Pulmonary Exacerbations [ Time Frame: Up to 2 years ]
    Annual exposure-adjusted incidence rate of pulmonary exacerbations.
  • Time to First Pulmonary Exacerbation [ Time Frame: Up to 2 years ]
  • Percentage of Subjects With Treatment Emergent Adverse Events [ Time Frame: From baseline up to 2.5 years ]
    Percentage of subjects with treatment-emergent adverse events (TEAEs): overall, by severity, by relatedness, by seriousness, and which occurred within 24 hours of Zemaira administration.
Original Secondary Outcome Measures  ICMJE
 (submitted: April 30, 2008)
  • Pulmonary Exacerbations [ Time Frame: ongoing ]
  • Pulmonary Function [ Time Frame: Quarterly ]
  • Antigenic and functional serum A1 - PI Levels [ Time Frame: Quarterly ]
  • Body mass index [ Time Frame: Quarterly ]
  • Quality of Life [ Time Frame: Yearly ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
Official Title  ICMJE An Open-label, Non-controlled, Multicenter, Multinational Study to Evaluate the Efficacy and Safety of Zemaira® Administration in Chronic Augmentation and Maintenance Therapy in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency Who Completed Clinical Study CE1226_4001
Brief Summary This study is a continuation of the placebo-controlled study CE1226_4001 (NCT00261833) to evaluate the efficacy and safety of Zemaira® intravenous (i.v). administration in subjects with emphysema due to alpha1-proteinase inhibitor deficiency. The long-term verification of a disease-modifying benefit of Zemaira® on the progression of emphysema will be assessed by volume-adjusted lung density, measured yearly by computed tomography (CT).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Emphysema
  • Alpha 1-proteinase Inhibitor Deficiency
Intervention  ICMJE Biological: Alpha1- proteinase inhibitor [human]
Lyophilized preparation of 60 mg/kg body weight intravenously once per week
Other Names:
  • Zemaira®
  • Respreeza®
Study Arms  ICMJE Experimental: Zemaira®
Intervention: Biological: Alpha1- proteinase inhibitor [human]
Publications *

*   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: December 10, 2013)
140
Original Estimated Enrollment  ICMJE
 (submitted: April 30, 2008)
100
Actual Study Completion Date  ICMJE September 2014
Actual Primary Completion Date September 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Subjects who have completed the 2-year treatment and observation period in the Phase 3/4 Zemaira® CE1226_4001 study (NCT00261833) and are willing to sign informed consent
  • Males, and non-pregnant, non-lactating females, whose screening pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator

Exclusion Criteria:

  • Individuals residing in the US
  • Current evidence of alcohol abuse or abuse of drugs such as barbiturates, benzodiazepines, amphetamines, cocaine, opioids, and cannabinoids
  • History of allergy, anaphylactic reaction, or severe systemic response to human plasma derived products, or known mannitol hypersensitivity, or history of prior adverse reaction to mannitol
  • Current tobacco smoker (smoking must be discontinued for at least 6 months prior to study participation)
  • Conditions or behaviors that interfere with attending scheduled study visits in the opinion of the investigator
  • History of non-compliance
  • Administration of any other experimental new drug or participation in an investigation of a marketed product
  • Inability to perform necessary study procedures
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 Australia,   Canada,   Czech Republic,   Denmark,   Estonia,   Finland,   Germany,   Ireland,   Poland,   Romania,   Sweden
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00670007
Other Study ID Numbers  ICMJE CE1226_3001
1466 ( Other Identifier: CSL Behring )
2007-007129-38 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party CSL Behring
Study Sponsor  ICMJE CSL Behring
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Program Director, Clinical R&D CSL Behring
PRS Account CSL Behring
Verification Date July 2016

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