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| ID | Type | Description | Link |
|---|---|---|---|
| 1449 | Other Identifier | CSL Behring | |
| 2005-003459-12 | EudraCT Number |
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This is a randomized, placebo-controlled, double-blind, multicenter phase III/IV study to compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to alpha1-proteinase inhibitor deficiency. The effect of Zemaira® on the progression of emphysema will be assessed by the decline of lung density, measured by computed tomography (CT).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Zemaira® | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Alpha1-proteinase inhibitor | Biological | 60 mg/kg body weight/week intravenous |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annual Rate of Change in Lung Density | As measured by centralized, standardized computer tomographic (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 rate of decline in each treatment group. | Over a 2-year period |
| Measure | Description | Time Frame |
|---|---|---|
| Annual Rate of Pulmonary Exacerbations | Primary diagnostic criteria for exacerbations were increased dyspnea, increased sputum volume, and increased sputum purulence. Supporting diagnostic criteria were upper respiratory tract infection, fever without other apparent cause, increased wheezing, and increased cough. For diagnosis, participants had to meet 2 of the 3 primary criteria or 1 primary criterion and 1 supporting criterion. The annual rate was based on the total number of exacerbations and the total number of participant study days for all participants in the specified analysis population and adjusted to 365.25 days. |
| Measure | Description | Time Frame |
|---|---|---|
| Baseline Lung Density at Total Lung Capacity (TLC) and Forced Residual Capacity (FRC) | Baseline |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Senior Director Immonology & Pulmonology, Clinical R&D | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Study Site | Denver | Colorado | 80206 | United States | ||
| Study Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30237305 | Derived | Greulich T, Chlumsky J, Wencker M, Vit O, Fries M, Chung T, Shebl A, Vogelmeier C, Chapman KR, McElvaney NG; RAPID Trial Group. Safety of biweekly alpha1-antitrypsin treatment in the RAPID programme. Eur Respir J. 2018 Nov 29;52(5):1800897. doi: 10.1183/13993003.00897-2018. Print 2018 Nov. | |
| 26026936 | Derived |
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Screening took place 1 to 4 weeks prior to the first dose of randomized investigational product (ie, either Zemaira® or placebo). A total of 208 participants were screened; 28 of these did not fulfill all eligibility criteria and were therefore screening failures.
This multicenter, multinational study enrolled participants at 28 study centers in Europe, North America, and Australia.
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| ID | Title | Description |
|---|---|---|
| FG000 | Zemaira® | Alpha1-proteinase inhibitor: 60 mg/kg body weight/week intravenous |
| FG001 | Placebo | Lyophilized preparation: 60 mg/kg body weight/week intravenous |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Placebo | Other | Lyophilized preparation: 60 mg/kg body weight/week intravenous |
|
| Over a 2-year period |
| Percent Change in FEV1 | Percent change from baseline to Month 24. | From baseline to 2 years |
| Time to First Pulmonary Exacerbation | Primary diagnostic criteria for exacerbations were increased dyspnea, increased sputum volume, and increased sputum purulence. Supporting diagnostic criteria were upper respiratory tract infection, fever without other apparent cause, increased wheezing, and increased cough. For diagnosis, participants had to meet 2 of the 3 primary criteria or 1 primary criterion and 1 supporting criterion. | Over a 2-year period |
| Change in Lung Density | Change from baseline to Month 24 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. | From baseline to 2 years |
| Change in Exercise Capacity | Exercise capacity was measured as distance walked, using the incremental shuttle walk test. Change from baseline to end of treatment (2 years) in exercise capacity was analysed using an analysis of covariance (ANCOVA). | From baseline to 2 years |
| Change in Patient-reported Symptoms | Patient-reported symptoms were measured using the symptoms score component of the St George's Respiratory Questionnaire (SGRQ). SGRQ scores range from 0 to 100, with higher scores indicating more limitations and negative values for change indicating improvement. Change from baseline to end of treatment (2 years) in SGRQ was analysed using an ANCOVA. | From baseline to 2 years |
| Frequency and Intensity of Adverse Events (AEs) | Number of participants with at least one AE, and the number of participants with mild, moderate or severe AEs. AE intensity was defined as mild (does not interfere with routine activities), moderate (interferes with routine activities), or severe (impossible to perform routine activities). | Over a 2-year period |
| Percent Change in Percent Predicted FEV1 | Percent change from baseline to Month 24. | From baseline to 2 years |
| Percent Change in FEV1 Divided by Forced Vital Capacity | Percent change from baseline to Month 24. | From baseline to 2 years |
| Percent Change in DLCO | Percent change from baseline to Month 24. | From baseline to 2 years |
| Duration of Pulmonary Exacerbations Relative to Treatment Duration | Defined as the percentage of total treatment duration across participants for 1) exacerbations overall, 2) antibiotic treatment for exacerbations, and 3) hospitalization for exacerbations. Primary diagnostic criteria for exacerbations were increased dyspnea, increased sputum volume, and increased sputum purulence. Supporting diagnostic criteria were upper respiratory tract infection, fever without other apparent cause, increased wheezing, and increased cough. For diagnosis, participants had to meet 2 of the 3 primary criteria or 1 primary criterion and 1 supporting criterion. | Over a 2-year period |
| Severity of Pulmonary Exacerbations | Defined as the number of participants requiring 1) antibiotic treatment for exacerbations, and 2) hospitalization for exacerbations. Primary diagnostic criteria for exacerbations were increased dyspnea, increased sputum volume, and increased sputum purulence. Supporting diagnostic criteria were upper respiratory tract infection, fever without other apparent cause, increased wheezing, and increased cough. For diagnosis, participants had to meet 2 of the 3 primary criteria or 1 primary criterion and 1 supporting criterion. Antibiotic treatment usage was reported by quarterly interval. | Over a 2-year period |
| Miami |
| Florida |
| 33136 |
| United States |
| Study Site | Hershey | Pennsylvania | 17033 | United States |
| Study Site | Tyler | Texas | 75708 | United States |
| Study Site | Darlinghurst | New South Wales | 2010 | Australia |
| Study Site | New Lambton | New South Wales | 2305 | Australia |
| Study Site | Brisbane | Queensland | 4066 | Australia |
| Study Site | Adelaide | South Australia | 5000 | Australia |
| Study Site | Nedlands | Western Australia | 6009 | Australia |
| Study Site | Fitzroy | 3065 | Australia |
| Study Site | Vancouver | British Columbia | V5Z4E1 | Canada |
| Study Site | Halifax | Nova Scotia | B3H3A7 | Canada |
| Study Site | Toronto | Ontario | M5T2S8 | Canada |
| Study Site | Prague | 14059 | Czechia |
| Study Site | Aarhus | 8000 | Denmark |
| Study Site | Hellerup | 2900 | Denmark |
| Study Site | Tartu | 51014 | Estonia |
| Study Site | Oulu | 90220 | Finland |
| Study Site | Berlin | 12200 | Germany |
| Study Site | Essen | 45239 | Germany |
| Study Site | Heidelberg | 69126 | Germany |
| Study Site | Nuremberg | 90419 | Germany |
| Study Site | Dublin | 9 | Ireland |
| Study Site | Krakow | 31-066 | Poland |
| Study Site | Warsaw | 01-138 | Poland |
| Study Site | Bucharest | 011026 | Romania |
| Study Site | Barnaul | Russia |
| Study Site | Malmö | 20502 | Sweden |
| Chapman KR, Burdon JG, Piitulainen E, Sandhaus RA, Seersholm N, Stocks JM, Stoel BC, Huang L, Yao Z, Edelman JM, McElvaney NG; RAPID Trial Study Group. Intravenous augmentation treatment and lung density in severe alpha1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial. Lancet. 2015 Jul 25;386(9991):360-8. doi: 10.1016/S0140-6736(15)60860-1. Epub 2015 May 27. |
| COMPLETED |
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| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Zemaira® | Alpha1-proteinase inhibitor: 60 mg/kg body weight/week intravenous |
| BG001 | Placebo | Lyophilized preparation: 60 mg/kg body weight/week intravenous |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Baseline percent predicted forced expiratory volume in 1 second (FEV1) | Mean | Standard Deviation | percentage |
| |||||||||||||||
| Baseline diffusion capacity of carbon monoxide (DLCO) | Mean | Standard Deviation | mL/min/mmHg |
| |||||||||||||||
| Number of participants with ZZ genotype (A1-PI deficiency), or SZ, Z / Null or Other genotype | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Annual Rate of Change in Lung Density | As measured by centralized, standardized computer tomographic (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 rate of decline in each treatment group. | All randomized participants with at least 1 valid CT scan. | Posted | Least Squares Mean | Standard Error | g/L per year | Over a 2-year period |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Annual Rate of Pulmonary Exacerbations | Primary diagnostic criteria for exacerbations were increased dyspnea, increased sputum volume, and increased sputum purulence. Supporting diagnostic criteria were upper respiratory tract infection, fever without other apparent cause, increased wheezing, and increased cough. For diagnosis, participants had to meet 2 of the 3 primary criteria or 1 primary criterion and 1 supporting criterion. The annual rate was based on the total number of exacerbations and the total number of participant study days for all participants in the specified analysis population and adjusted to 365.25 days. | All participants with A1-PI deficiency who were included in the study and randomized. | Posted | Number | 95% Confidence Interval | exacerbations per participant year | Over a 2-year period |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change in FEV1 | Percent change from baseline to Month 24. | All participants with A1-PI deficiency who were included in the study, randomized, and had a baseline and at least one endpoint assessment available. | Posted | Least Squares Mean | Standard Error | percent change | From baseline to 2 years |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Time to First Pulmonary Exacerbation | Primary diagnostic criteria for exacerbations were increased dyspnea, increased sputum volume, and increased sputum purulence. Supporting diagnostic criteria were upper respiratory tract infection, fever without other apparent cause, increased wheezing, and increased cough. For diagnosis, participants had to meet 2 of the 3 primary criteria or 1 primary criterion and 1 supporting criterion. | All participants with A1-PI deficiency who were included in the study and randomized. | Posted | Median | 95% Confidence Interval | Years | Over a 2-year period |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Lung Density | Change from baseline to Month 24 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. | All participants with A1-PI deficiency who were included in the study, randomized, and had a baseline and at least 1 endpoint assessment available. | Posted | Least Squares Mean | Standard Error | g/L | From baseline to 2 years |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Exercise Capacity | Exercise capacity was measured as distance walked, using the incremental shuttle walk test. Change from baseline to end of treatment (2 years) in exercise capacity was analysed using an analysis of covariance (ANCOVA). | All participants with A1-PI deficiency who were included in the study, randomized, and had a baseline and at least one endpoint assessment available. | Posted | Least Squares Mean | Standard Error | metre | From baseline to 2 years |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Patient-reported Symptoms | Patient-reported symptoms were measured using the symptoms score component of the St George's Respiratory Questionnaire (SGRQ). SGRQ scores range from 0 to 100, with higher scores indicating more limitations and negative values for change indicating improvement. Change from baseline to end of treatment (2 years) in SGRQ was analysed using an ANCOVA. | All participants with A1-PI deficiency who were included in the study, randomized, and had a baseline and at least one endpoint assessment available. | Posted | Least Squares Mean | Standard Error | units on a scale | From baseline to 2 years |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Frequency and Intensity of Adverse Events (AEs) | Number of participants with at least one AE, and the number of participants with mild, moderate or severe AEs. AE intensity was defined as mild (does not interfere with routine activities), moderate (interferes with routine activities), or severe (impossible to perform routine activities). | All participants receiving at least 1 infusion of either Zemaira® or placebo. | Posted | Number | participants | Over a 2-year period |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change in Percent Predicted FEV1 | Percent change from baseline to Month 24. | All participants with A1-PI deficiency who were included in the study, randomized, and had a baseline and at least one endpoint assessment available. | Posted | Least Squares Mean | Standard Error | percent change | From baseline to 2 years |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change in FEV1 Divided by Forced Vital Capacity | Percent change from baseline to Month 24. | All participants with A1-PI deficiency who were included in the study, randomized, and had a baseline and at least one endpoint assessment available. | Posted | Least Squares Mean | Standard Error | percent change | From baseline to 2 years |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change in DLCO | Percent change from baseline to Month 24. | All participants with A1-PI deficiency who were included in the study, randomized, and had a baseline and at least one endpoint assessment available. | Posted | Least Squares Mean | Standard Error | percent change | From baseline to 2 years |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Duration of Pulmonary Exacerbations Relative to Treatment Duration | Defined as the percentage of total treatment duration across participants for 1) exacerbations overall, 2) antibiotic treatment for exacerbations, and 3) hospitalization for exacerbations. Primary diagnostic criteria for exacerbations were increased dyspnea, increased sputum volume, and increased sputum purulence. Supporting diagnostic criteria were upper respiratory tract infection, fever without other apparent cause, increased wheezing, and increased cough. For diagnosis, participants had to meet 2 of the 3 primary criteria or 1 primary criterion and 1 supporting criterion. | All participants with A1-PI deficiency who were included in the study and randomized. | Posted | Mean | Standard Deviation | percentage of total treatment duration | Over a 2-year period |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Severity of Pulmonary Exacerbations | Defined as the number of participants requiring 1) antibiotic treatment for exacerbations, and 2) hospitalization for exacerbations. Primary diagnostic criteria for exacerbations were increased dyspnea, increased sputum volume, and increased sputum purulence. Supporting diagnostic criteria were upper respiratory tract infection, fever without other apparent cause, increased wheezing, and increased cough. For diagnosis, participants had to meet 2 of the 3 primary criteria or 1 primary criterion and 1 supporting criterion. Antibiotic treatment usage was reported by quarterly interval. | All participants with A1-PI deficiency who were included in the study and randomized. | Posted | Number | participants | Over a 2-year period |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Baseline Lung Density at Total Lung Capacity (TLC) and Forced Residual Capacity (FRC) | Posted | Mean | Standard Deviation | g/L | Baseline |
|
|
For the duration of the study (25 months; 1 month screening period, 24 months treatment period).
The randomized, double-blind investigational product (Zemaira® or placebo) was administered as 60 mg once per week for 24 months. In exceptional cases, a dose of 120 mg could be given to cover a 2-week period. The safety population comprised all participants receiving at least 1 infusion of either Zemaira® or placebo.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Zemaira® | Alpha1-proteinase inhibitor: 60 mg/kg body weight/week intravenous | 28 | 93 | 90 | 93 | ||
| EG001 | Placebo | Lyophilized preparation: 60 mg/kg body weight/week intravenous | 28 | 87 | 84 | 87 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Graft infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Lung neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Systematic Assessment |
| |
| Bladder cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Systematic Assessment |
| |
| Breast cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Systematic Assessment |
| |
| Breast cancer metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Systematic Assessment |
| |
| Lung neoplasm malignant | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Systematic Assessment |
| |
| Parathyroid tumour benign | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Gastroesophageal reflux disease | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Adhesion | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Aortic aneurysm repair | Surgical and medical procedures | MedDRA 15.0 | Systematic Assessment |
| |
| Tonsillectomy | Surgical and medical procedures | MedDRA 15.0 | Systematic Assessment |
| |
| Transurethral prostatectomy | Surgical and medical procedures | MedDRA 15.0 | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Eye inflammation | Eye disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA 15.0 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
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CSL agreements and restrictions on publishing may vary with individual investigators; however, CSL will not prohibit any investigator from publishing. CSL supports the publication of results from all centers of a multi-center trial and generally requires that reports based on single-site data not precede the primary publication of the entire clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Disclosure Manager | CSL Behring | Use email contact | clinicaltrials@cslbehring.com |
| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| D004646 | Emphysema |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| D000515 | alpha 1-Antitrypsin |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D015843 | Serpins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000209 | Acute-Phase Proteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000510 | Alpha-Globulins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
Not provided
Not provided
| Male |
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| SZ |
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| Z / Null |
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| Other |
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| FRC |
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| No |
| Superiority or Other |
| Analysis of the annual rate of change in lung density (TLC) was a linear mixed model with country, time since baseline treatment, and treatment-by-time interaction as fixed effects and participant and participant-by-time interaction as random coefficients at a 1-sided significance level of 0.025. | 95% confidence interval | 0.017 | A 1-sided P-value less than 0.025 and a positive estimate of the treatment difference Zemaira® minus placebo (ie, the lower bound of the 95% CI being greater than zero) will indicate superiority of Zemaira® compared with Placebo. | Difference in lung density(adjusted P15) | 0.740 | 2-Sided | 95 | 0.059 | 1.420 | A mixed model was used to estimate the 95% CI for the difference (Zemaira® - placebo) in annual lung density decline. | No | Superiority or Other |
| Analysis of the annual rate of change in lung density (FRC) was a linear mixed model with country, time since baseline treatment, and treatment-by-time interaction as fixed effects and participant and participant-by-time interaction as random coefficients at a 1-sided significance level of 0.025. | 95% confidence interval | 0.090 | A 1-sided P-value less than 0.025 and a positive estimate of the treatment difference Zemaira® minus placebo (ie, the lower bound of the 95% CI being greater than zero) will indicate superiority of Zemaira® compared with Placebo. | Difference in lung density(adjusted P15) | 0.478 | 2-Sided | 95 | -0.223 | 1.180 | A mixed model was used to estimate the 95% CI for the difference (Zemaira® - placebo) in annual lung density decline. | No | Superiority or Other |
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