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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
127
Weekly ARALAST infusions for 2 years, dose and mode of administration as prescribed by the physician
Adupa Rao, MD
San Marino, California, United States
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline to 24 months
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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
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Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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
Time frame: Baseline to 24 Months