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Study terminated early due to Aralast being phased out of the market.
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The primary objectives of this Phase 4, open label, prospective U.S. surveillance study are to evaluate the health outcomes of Alpha 1-Antitrypsin (AAT)-deficient subjects who are initiating treatment with ARALAST on patient-related outcomes (PRO), i.e., health-related quality of life (HRQoL), healthcare resource utilization (HCRU), and various laboratory analyses to evaluate the safety of long-term administration of ARALAST.
Up to 120 subjects will be enrolled and assessed for HRQoL and HCRU at baseline and every 6-months thereafter, for 2 years. A subset of subjects will be enrolled into the blood draw portion of the study, which will also include assessments of antibodies to ARALAST, and chemistry panel. Subjects will be treated according to the prescribing (attending) physician's instructions based on the prescribing information given in the ARALAST package insert.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ARALAST Alpha1-Proteinase Inhibitor | Drug | Weekly ARALAST infusions for 2 years, dose and mode of administration as prescribed by the physician |
| Measure | Description | Time Frame |
|---|---|---|
| HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Screening to ≤ 6 Months |
| HRQoL 'Role Limitation Due to Physical Health (RP)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Screening to ≤ 6 Months |
| HRQoL 'Bodily Pain (BP)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Screening to ≤ 6 Months |
| HRQoL 'General Health (GH)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. |
| Measure | Description | Time Frame |
|---|---|---|
| Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits' | Number of participants with indicated number of ER visits (0, 1, 2, 3, ≥4 ER visits per participant) during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Baseline to 24 Months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Adupa Rao, MD | San Marino | California | 91108 | United States |
127 participants enrolled in the study, of these 126 participated in the health related quality of life (HRQoL) and healthcare resource utilization (HCRU) portion, 61 consented to the blood draw portion, and 66 only consented to the Quality of Life portion
This was a surveillance study conducted by Coram, Inc., in locations throughout the United States, and supervised by Baxter. Enrollment began in June 2006 and the study was terminated early, in December 2008, due to Aralast being phased out of the market.
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| ID | Title | Description |
|---|---|---|
| FG000 | Overall Study | Participants were treated with ARALAST according to dose and frequency of infusions recommended by their physician. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | Overall Study |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median |
| 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 | HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
|
Throughout the entire study period (2 years, 7 months)
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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 | Overall Study |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bradycardia | Cardiac disorders | MedDRA (Unspecified) |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | MedDRA (Unspecified) |
Study was terminated early due to Aralast being phased out of the market.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
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| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| Screening to ≤ 6 Months |
| HRQoL 'Vitality (VT)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Screening to ≤ 6 Months |
| HRQoL 'Social Functioning (SF)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Screening to ≤ 6 Months |
| HRQoL 'Role Limitation Due to Emotional Problems (RE)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Screening to ≤ 6 Months |
| HRQoL 'Mental Health (MH)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Screening to ≤ 6 Months |
| HRQoL 'Physical Component Score (PCS)' From Baseline to ≤6 Months | SF-36 scores for baseline (screening) versus the period from baseline to ≤6 Months. The PCS is a summary scale of the dimensions physical functioning, role physical, bodily pain, and general health. The component score is normalized to a standard population. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. | Screening to ≤ 6 Months |
| HRQoL 'Mental Component Score (MCS)' From Baseline to ≤6 Months | SF-36 scores for baseline (screening) versus the period from baseline to ≤6 Months. The MCS is a summary scale of the dimensions vitality, social functioning, role emotional, and mental health Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. | Screening to ≤ 6 Months |
| HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months | SF-36 Scores- baseline thru 12 months, where data was available. Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The Data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Baseline to 12 months |
| HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months | SF-36 Scores- baseline thru 12 months, where data was available. Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The Data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Baseline to 12 months |
| HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months | SF-36 Scores- baseline thru 24 months, where data was available. Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The Data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Baseline to 24 months |
| Healthcare Resource Utilization (HCRU) 'Mean Number of Emergency Room (ER) Visits' |
Mean number of ER visits one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) |
| One year prior to baseline to 24 months post-baseline |
| Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations' | Number of participants with indicated number of hospitalizations during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Baseline to 24 Months |
| Healthcare Resource Utilization (HCRU) 'Mean Length of Stay (LOS) in Hospital' | Mean LOS during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Baseline to 24 months |
| Healthcare Resource Utilization (HCRU) 'Number of Participants Taking Antibiotics' | Number of participants taking antibiotics one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | One year prior to baseline to 24 months post-baseline |
| Healthcare Resource Utilization (HCRU) 'Number of Antibiotic Courses' | Number of antibiotic courses (i.e. number of antibiotic prescriptions) one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | One year prior to baseline to 24 months post-baseline |
| Healthcare Resource Utilization (HCRU) 'Number of Participants Receiving Steroid Pulse Courses' | Number of participants receiving steroid pulse courses (i.e. number of steroid prescriptions) one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | One year prior to baseline to 24 months post-baseline |
| Healthcare Resource Utilization (HCRU) 'Number of Steroid Pulse Courses' | Number of steroid pulse courses (i.e. number of steroid prescriptions) one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | One year prior to baseline to 24 months post-baseline |
| Hepatic Chemistry Parameters: Change From Baseline/Screening | Summary of changes in hepatic (total bilirubin, aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase) parameters from screening/baseline through each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Baseline to 24 months |
| Renal and Hepatic Chemistry Parameters: Change From Baseline/Screening | Summary of changes in hepatic (total bilirubin) and renal (Blood urea nitrogen (BUN), creatinine) parameters from screening/baseline through each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Baseline to 24 months |
| ARALAST Antibody Titers: Participants With at Least 2-Dilution Step Increases From Screening | All IgG and IgM titers at screening were ≤ 4. A 2-dilution step increase was defined as follows: • The titer at each 6-month visit must be ≥ 4 when the screening titer = 0 • Each 6-month visit titer / screening titer should be ≥ 4. 6 month window periods are: baseline to ≤6 months, >6 months to ≤12 months, >12 months to ≤18 months, and >18 months to ≤24 months | Baseline to 24 Months |
| Withdrawal by Subject |
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| Terminated by sponsor/ homecare provider |
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| Physician Decision |
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| Adverse event - transient |
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| Liver transplant |
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| Insurance (homecare provider) change |
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| Change to different product |
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| Due to termination of the study |
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| years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Count of Participants | Participants |
|
| Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) | The SF-36 is a validated instrument measuring health-related quality of life across multiple disease states. It has 36 questions with 8 subscale scores and 2 summary scores (1) physical component summary score (PCS)=physical functioning, role-physical, bodily pain, and general health; (2) mental component summary score (MCS)=vitality, social functioning, role-emotional, and mental health. There is no total overall score; scoring is done for both subscores and summary scores. For subscores and summary scores, 0 =worst score (or quality of life) and 100=best score. | Mean | Standard Deviation | Scores on a scale |
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| Baseline to ≤6 Months |
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| Primary | HRQoL 'Role Limitation Due to Physical Health (RP)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
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| Primary | HRQoL 'Bodily Pain (BP)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
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| Primary | HRQoL 'General Health (GH)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
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| Primary | HRQoL 'Vitality (VT)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
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| Primary | HRQoL 'Social Functioning (SF)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
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| Primary | HRQoL 'Role Limitation Due to Emotional Problems (RE)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
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| Primary | HRQoL 'Mental Health (MH)' From Baseline to ≤6 Months | Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
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| Primary | HRQoL 'Physical Component Score (PCS)' From Baseline to ≤6 Months | SF-36 scores for baseline (screening) versus the period from baseline to ≤6 Months. The PCS is a summary scale of the dimensions physical functioning, role physical, bodily pain, and general health. The component score is normalized to a standard population. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
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| Primary | HRQoL 'Mental Component Score (MCS)' From Baseline to ≤6 Months | SF-36 scores for baseline (screening) versus the period from baseline to ≤6 Months. The MCS is a summary scale of the dimensions vitality, social functioning, role emotional, and mental health Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. | Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Screening to ≤ 6 Months |
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| Primary | HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months | SF-36 Scores- baseline thru 12 months, where data was available. Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The Data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤12 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Baseline to 12 months |
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| Primary | HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months | SF-36 Scores- baseline thru 12 months, where data was available. Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The Data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤18 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Baseline to 12 months |
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| Primary | HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months | SF-36 Scores- baseline thru 24 months, where data was available. Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The Data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000. | Participants with baseline and participating during the period from baseline to ≤24 months data for HRQoL | Posted | Mean | Standard Deviation | Scores on a scale | Baseline to 24 months |
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| Secondary | Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits' | Number of participants with indicated number of ER visits (0, 1, 2, 3, ≥4 ER visits per participant) during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Participants with data available during each window period | Posted | Number | Participants | Baseline to 24 Months |
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| Secondary | Healthcare Resource Utilization (HCRU) 'Mean Number of Emergency Room (ER) Visits' | Mean number of ER visits one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Participants with data available during each window period | Posted | Mean | Standard Deviation | ER visits per time period | One year prior to baseline to 24 months post-baseline |
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| Secondary | Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations' | Number of participants with indicated number of hospitalizations during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Participants with data available during each window period | Posted | Number | Participants | Baseline to 24 Months |
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| Secondary | Healthcare Resource Utilization (HCRU) 'Mean Length of Stay (LOS) in Hospital' | Mean LOS during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Participants with data available during each window period | Posted | Mean | Standard Deviation | Days | Baseline to 24 months |
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| Secondary | Healthcare Resource Utilization (HCRU) 'Number of Participants Taking Antibiotics' | Number of participants taking antibiotics one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Participants with data available during each window period | Posted | Number | Participants | One year prior to baseline to 24 months post-baseline |
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| Secondary | Healthcare Resource Utilization (HCRU) 'Number of Antibiotic Courses' | Number of antibiotic courses (i.e. number of antibiotic prescriptions) one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Participants with data available during each window period | Posted | Mean | Standard Deviation | Antibiotic courses | One year prior to baseline to 24 months post-baseline |
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| Secondary | Healthcare Resource Utilization (HCRU) 'Number of Participants Receiving Steroid Pulse Courses' | Number of participants receiving steroid pulse courses (i.e. number of steroid prescriptions) one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Participants with data available during each window period | Posted | Number | Participants | One year prior to baseline to 24 months post-baseline |
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|
|
| Secondary | Healthcare Resource Utilization (HCRU) 'Number of Steroid Pulse Courses' | Number of steroid pulse courses (i.e. number of steroid prescriptions) one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Participants with data available during each window period | Posted | Mean | Standard Deviation | Steroid Pulse Courses | One year prior to baseline to 24 months post-baseline |
|
|
|
| Secondary | Hepatic Chemistry Parameters: Change From Baseline/Screening | Summary of changes in hepatic (total bilirubin, aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase) parameters from screening/baseline through each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Subjects who participated in the blood draws with data available during each window period | Posted | Median | Full Range | U/L | Baseline to 24 months |
|
|
|
| Secondary | Renal and Hepatic Chemistry Parameters: Change From Baseline/Screening | Summary of changes in hepatic (total bilirubin) and renal (Blood urea nitrogen (BUN), creatinine) parameters from screening/baseline through each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months) | Subjects who participated in the blood draws with data available during each window period | Posted | Median | Full Range | mg/dL | Baseline to 24 months |
|
|
|
| Secondary | ARALAST Antibody Titers: Participants With at Least 2-Dilution Step Increases From Screening | All IgG and IgM titers at screening were ≤ 4. A 2-dilution step increase was defined as follows: • The titer at each 6-month visit must be ≥ 4 when the screening titer = 0 • Each 6-month visit titer / screening titer should be ≥ 4. 6 month window periods are: baseline to ≤6 months, >6 months to ≤12 months, >12 months to ≤18 months, and >18 months to ≤24 months | Subjects who participated in the blood draws with data available during each window period | Posted | Number | Participants | Baseline to 24 Months |
|
|
|
| 39 |
| 127 |
| 97 |
| 127 |
| Cardiac failure congestive | Cardiac disorders | MedDRA (Unspecified) |
|
| Cardio-respiratory arrest | Cardiac disorders | MedDRA (Unspecified) |
|
| Myocardial infarction | Cardiac disorders | MedDRA (Unspecified) |
|
| Colon gangrene | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| Gastric ulcer | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| Vomiting | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| Chest pain | General disorders | MedDRA (Unspecified) |
|
| Fatigue | General disorders | MedDRA (Unspecified) |
|
| Hernia | General disorders | MedDRA (Unspecified) |
|
| Hepatic failure | Hepatobiliary disorders | MedDRA (Unspecified) |
|
| Gastroenteritis | Infections and infestations | MedDRA (Unspecified) |
|
| Pneumonia | Infections and infestations | MedDRA (Unspecified) |
|
| Pseudomonas infection | Infections and infestations | MedDRA (Unspecified) |
|
| Respiratory tract infection | Infections and infestations | MedDRA (Unspecified) |
|
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA (Unspecified) |
|
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| Disorientation | Nervous system disorders | MedDRA (Unspecified) |
|
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) |
|
| Peripheral artery aneurysm | Vascular disorders | MedDRA (Unspecified) |
|
| Fatigue | General disorders | MedDRA (Unspecified) |
|
| Infusion related reaction | General disorders | MedDRA (Unspecified) |
|
| Hypersensitivity | Immune system disorders | MedDRA (Unspecified) |
|
| Influenza | Infections and infestations | MedDRA (Unspecified) |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA (Unspecified) |
|
| Oral fungal infection | Infections and infestations | MedDRA (Unspecified) |
|
| Respiratory tract infection | Infections and infestations | MedDRA (Unspecified) |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (Unspecified) |
|
| Headache | Nervous system disorders | MedDRA (Unspecified) |
|
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) |
|
| Elective procedure | Surgical and medical procedures | MedDRA (Unspecified) |
|
| Poor venous access | Vascular disorders | MedDRA (Unspecified) |
|
Baxter's agreements with PIs vary per individual PI, but contain common elements. Baxter may require a review of results communications (e.g., for confidential information & intellectual property) ≥45 days prior to submission or communication for review and comment. Baxter reserves the right to remove all confidential information from all publications.
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D004646 | Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
|
| Bodily Pain (BP) |
|
| General Health (GH) |
|
| Vitality (VT) |
|
| Social Functioning (SF) |
|
| Role Limitation Due to Emotional Problems (RE) |
|
| Mental Health (MH) |
|
| Physical Component Score (PCS) |
|
| Mental Component Score (MCS) |
|
| Role Limitation Due to Physical Health (RP) |
|
| Bodily Pain (BP) |
|
| General Health (GH) |
|
| Vitality (VT) |
|
| Social Functioning (SF) |
|
| Role Limitation Due to Emotional Problems (RE) |
|
| Mental Health (MH) |
|
| Physical Component Score (PCS) |
|
| Mental Component Score (MCS) |
|
| Role Limitation Due to Physical Health (RP) |
|
| Bodily Pain (BP) |
|
| General Health (GH) |
|
| Vitality (VT) |
|
| Social Functioning (SF) |
|
| Role Limitation Due to Emotional Problems (RE) |
|
| Mental Health (MH) |
|
| Physical Component Score (PCS) |
|
| Mental Component Score (MCS) |
|
| 1 ER visits |
|
| 2 ER visits |
|
| 3 ER visits |
|
| ≥4 ER visits |
|
| 1 Hospitalizations |
|
| 2 Hospitalizations |
|
| 3 Hospitalizations |
|
| ≥4 Hospitalizations |
|
| Oral antibiotic courses |
|
| I.V. antibiotic courses |
|
| Oral steroid pulse courses |
|
| I.V. steroid pulse courses |
|
| ALT |
|
| AST |
|
| Total bilirubin |
|
| BUN |
|
| IgM |
|