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 |
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Recruitment Status :
Completed
First Posted : May 1, 2008
Results First Posted : July 12, 2016
Last Update Posted : August 15, 2016
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Sponsor:
CSL Behring
Information provided by (Responsible Party):
CSL Behring
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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).
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Emphysema Alpha 1-proteinase Inhibitor Deficiency | Biological: Alpha1- proteinase inhibitor [human] | Phase 4 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 140 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official 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 |
| Study Start Date : | April 2008 |
| Actual Primary Completion Date : | September 2014 |
| Actual Study Completion Date : | September 2014 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Alpha-1 antitrypsin deficiency
| Arm | Intervention/treatment |
|---|---|
| Experimental: Zemaira® |
Biological: Alpha1- proteinase inhibitor [human]
Lyophilized preparation of 60 mg/kg body weight intravenously once per week
Other Names:
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Primary Outcome Measures :
- 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 Outcome Measures :
- 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.
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
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
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 ClinicalTrials.gov identifier (NCT number): NCT00670007
Locations
| Australia, South Australia | |
| Study Site | |
| Adelaide, South Australia, Australia, 5000 | |
| Australia, Victoria | |
| Study Site | |
| Fitzroy, Victoria, Australia, 3065 | |
| Australia | |
| Study Site | |
| Darlinghurst, Australia, 2010 | |
| Study Site | |
| Nedlands, Australia, 6009 | |
| Study Site | |
| New Lambton, Australia, 2305 | |
| Canada, British Columbia | |
| Study Site | |
| Vancouver, British Columbia, Canada, V6J1S3 | |
| Canada, Nova Scotia | |
| Study Site | |
| Halifax, Nova Scotia, Canada, B3H 3A7 | |
| Canada, Ontario | |
| Study Site | |
| Toronto, Ontario, Canada, M5T 2S8 | |
| Czech Republic | |
| Study Site | |
| Praha 4 - Krc, Czech Republic, 14059 | |
| Denmark | |
| Study Site | |
| Arhus C, Denmark, 8000 | |
| Study Site | |
| Hellerup, Denmark, 2900 | |
| Estonia | |
| Study Site | |
| Tartu, Estonia, 51014 | |
| Finland | |
| Study Site | |
| Oulu, Finland, 90220 | |
| Germany | |
| Study Site | |
| Essen, Germany, 45239 | |
| Study Site | |
| Heidelberg, Germany, 69126 | |
| Study Site | |
| Nürnberg, Germany, 90419 | |
| Ireland | |
| Study Site | |
| Dublin, Ireland, 9 | |
| Poland | |
| Study Site | |
| Krakow, Poland, 31-066 | |
| Study Site | |
| Warsaw, Poland, 01-138 | |
| Romania | |
| Study Site | |
| Bucuresti, Romania, 011026 | |
| Sweden | |
| Study Site | |
| Malmo, Sweden, 20502 | |
Sponsors and Collaborators
CSL Behring
Investigators
| Study Director: | Program Director, Clinical R&D | CSL Behring |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | CSL Behring |
| ClinicalTrials.gov Identifier: | NCT00670007 |
| Other Study ID Numbers: |
CE1226_3001 1466 ( Other Identifier: CSL Behring ) 2007-007129-38 ( EudraCT Number ) |
| First Posted: | May 1, 2008 Key Record Dates |
| Results First Posted: | July 12, 2016 |
| Last Update Posted: | August 15, 2016 |
| Last Verified: | July 2016 |
Keywords provided by CSL Behring:
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Alpha1-proteinase inhibitor deficiency Chronic augmentation and maintenance therapy Emphysema Emphysema due to Alpha 1-proteinase inhibitor deficiency |
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency Pulmonary Emphysema Emphysema Pathologic Processes Pulmonary Disease, Chronic Obstructive Lung Diseases, Obstructive Lung Diseases Respiratory Tract Diseases Liver Diseases |
Digestive System Diseases Genetic Diseases, Inborn Subcutaneous Emphysema Protease Inhibitors Alpha 1-Antitrypsin Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Trypsin Inhibitors Serine Proteinase Inhibitors |