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Completed

NCT Number: NCT00670007

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

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).

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Key information

Conditions

Emphysema Alpha 1-proteinase Inhibitor Deficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Liver Diseases Lung Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Respiratory Tract Diseases Subcutaneous Emphysema alpha 1-Antitrypsin Deficiency

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Study Site, Adelaide, South Australia, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Alpha1- proteinase inhibitor [human]

Biological

Lyophilized preparation of 60 mg/kg body weight intravenously once per week

Other names: Zemaira®, Respreeza®

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. Percent Change in Lung Function as Measured by Forced Expiratory Volume in 1 Second (FEV1)

    Time frame: From baseline up to 2 years

  5. Percent Change in Lung Function as Measured by Ratio of FEV1/FVC (Forced Vital Capacity)

    Time frame: From baseline up to 2 years

  6. Percent Change in Lung Function as Measured by Percent Predicted FEV1

    Time frame: From baseline up to 2 years

  7. Number of Subjects With Pulmonary Exacerbations

    Time frame: Up to 2 years

  8. Annual Rate in Subject Years of Pulmonary Exacerbations

    Time frame: Up to 2 years

    Annual exposure-adjusted incidence rate of pulmonary exacerbations.

  9. Time to First Pulmonary Exacerbation

    Time frame: Up to 2 years

  10. 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.

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

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

Important dates

Study start
2008
Primary completion
2014
Study completion
2014
First posted
May 1, 2008
Registry last updated
Aug 15, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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