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Aralast alpha1-proteinase Inhibitor Surveillance Study

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ClinicalTrials.gov Identifier: NCT00313144
Recruitment Status : Terminated (Study terminated early due to Aralast being phased out of the market.)
First Posted : April 12, 2006
Results First Posted : August 26, 2011
Last Update Posted : February 12, 2018
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Alpha1-antitrypsin Deficiency
Intervention Drug: ARALAST Alpha1-Proteinase Inhibitor
Enrollment 127
Recruitment Details 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.
Pre-assignment Details 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
Arm/Group Title Overall Study
Hide Arm/Group Description Participants were treated with ARALAST according to dose and frequency of infusions recommended by their physician.
Period Title: Overall Study
Started 127
Completed 17
Not Completed 110
Reason Not Completed
Discontinue Aralast ≥4 consecutive doses             12
Death             7
Lost to Follow-up             5
Withdrawal by Subject             4
Terminated by sponsor/ homecare provider             4
Physician Decision             4
Adverse event - transient             5
Liver transplant             1
Insurance (homecare provider) change             1
Change to different product             1
Due to termination of the study             66
Arm/Group Title Overall Study
Hide Arm/Group Description [Not Specified]
Overall Number of Baseline Participants 127
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 127 participants
52.3
(21.2 to 84.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 127 participants
Female
68
  53.5%
Male
59
  46.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 127 participants
127
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126)   [1] 
Mean (Standard Deviation)
Unit of measure:  Scores on a scale
Number Analyzed 127 participants
Physical Functioning (PF) 31.612  (11.678)
Role Limitation Due to Physical Health (RP) 37.281  (11.871)
Bodily Pain (BP) 46.368  (11.116)
General Health (GH) 37.351  (10.120)
Vitality (VT) 42.279  (11.209)
Social Functioning (SF) 41.699  (14.069)
Role Limitation Due to Emotional Problems (RE) 48.474  (10.687)
Mental Health (MH) 47.326  (11.358)
Physical Component Score (PCS) 34.150  (11.263)
Mental Component Score (MCS) 50.340  (11.897)
[1]
Measure Description: 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.
1.Primary Outcome
Title HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
31.958  (11.291) 33.129  (11.527)
2.Primary Outcome
Title HRQoL 'Role Limitation Due to Physical Health (RP)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
37.631  (11.819) 38.139  (11.338)
3.Primary Outcome
Title HRQoL 'Bodily Pain (BP)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
46.474  (10.895) 48.950  (11.579)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Baseline, Baseline to ≤6 Months
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.044
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
4.Primary Outcome
Title HRQoL 'General Health (GH)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
37.365  (10.038) 38.737  (10.490)
5.Primary Outcome
Title HRQoL 'Vitality (VT)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
42.578  (11.164) 45.081  (10.916)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Baseline, Baseline to ≤6 Months
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.009
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
6.Primary Outcome
Title HRQoL 'Social Functioning (SF)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
42.340  (14.060) 45.581  (12.780)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Baseline, Baseline to ≤6 Months
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.005
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
7.Primary Outcome
Title HRQoL 'Role Limitation Due to Emotional Problems (RE)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
48.361  (10.972) 47.921  (10.855)
8.Primary Outcome
Title HRQoL 'Mental Health (MH)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
47.457  (10.711) 50.007  (9.480)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Baseline, Baseline to ≤6 Months
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.006
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
9.Primary Outcome
Title HRQoL 'Physical Component Score (PCS)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
34.453  (10.957) 35.826  (10.881)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Baseline, Baseline to ≤6 Months
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.044
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
10.Primary Outcome
Title HRQoL 'Mental Component Score (MCS)' From Baseline to ≤6 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 106
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
50.464  (11.713) 52.378  (10.639)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Baseline, Baseline to ≤6 Months
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.046
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
11.Primary Outcome
Title HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤12 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months >6 Months to ≤12 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 65 65 65
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Physical Functioning (PF) 34.822  (11.173) 35.632  (11.251) 34.143  (11.914)
Role Limitation Due to Physical Health (RP) 40.539  (11.627) 39.484  (11.615) 38.542  (13.061)
Bodily Pain (BP) 47.203  (11.375) 50.227  (11.267) 47.788  (13.139)
General Health (GH) 38.853  (10.197) 39.990  (10.013) 40.026  (11.720)
Vitality (VT) 44.646  (11.546) 45.703  (11.516) 44.742  (11.058)
Social Functioning (SF) 44.683  (13.864) 47.200  (11.799) 43.927  (14.384)
Role Limitation Due to Emotional Problems (RE) 48.403  (11.214) 48.583  (10.590) 48.942  (12.011)
Mental Health (MH) 47.365  (11.040) 50.311  (8.962) 49.488  (11.705)
Physical Component Score (PCS) 37.346  (10.906) 37.890  (10.499) 36.272  (11.238)
Mental Component Score (MCS) 50.442  (11.896) 52.508  (10.354) 51.854  (12.088)
12.Primary Outcome
Title 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
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤18 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 47 47 47 47
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Physical Functioning (PF) 34.197  (11.375) 34.958  (11.048) 33.301  (11.764) 33.839  (12.546)
Role Limitation Due to Physical Health (RP) 40.492  (12.310) 39.033  (11.686) 37.887  (12.964) 40.336  (12.324)
Bodily Pain (BP) 47.525  (12.023) 50.726  (10.942) 48.990  (13.177) 50.537  (11.789)
General Health (GH) 38.684  (10.164) 39.718  (9.697) 39.475  (11.435) 39.586  (10.848)
Vitality (VT) 45.714  (11.843) 46.245  (10.807) 45.249  (11.002) 45.648  (10.901)
Social Functioning (SF) 45.825  (14.089) 47.218  (11.665) 44.781  (14.110) 45.013  (14.127)
Role Limitation Due to Emotional Problems (RE) 49.014  (11.497) 48.187  (11.158) 48.022  (13.331) 47.360  (11.930)
Mental Health (MH) 48.390  (11.740) 50.187  (9.347) 49.229  (12.562) 48.569  (11.902)
Physical Component Score (PCS) 36.803  (11.105) 37.655  (10.609) 36.171  (10.848) 38.060  (11.450)
Mental Component Score (MCS) 51.885  (12.215) 52.575  (10.846) 51.843  (13.323) 50.814  (12.729)
13.Primary Outcome
Title 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
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with baseline and participating during the period from baseline to ≤24 months data for HRQoL
Arm/Group Title Baseline Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 21 21 21 21 21
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Physical Functioning (PF) 36.489  (11.174) 35.988  (10.874) 34.585  (12.241) 35.487  (12.782) 33.883  (12.836)
Role Limitation Due to Physical Health (RP) 43.092  (13.267) 42.509  (11.390) 40.643  (13.245) 42.276  (11.577) 41.576  (12.841)
Bodily Pain (BP) 52.036  (13.330) 53.988  (11.695) 52.559  (14.045) 56.041  (10.951) 51.674  (12.214)
General Health (GH) 38.452  (11.496) 40.744  (10.222) 39.632  (11.720) 40.245  (11.852) 38.293  (11.171)
Vitality (VT) 47.036  (11.949) 48.969  (10.116) 49.118  (10.187) 47.036  (9.797) 47.631  (10.705)
Social Functioning (SF) 46.980  (16.418) 48.538  (12.026) 44.123  (14.361) 46.980  (13.756) 48.019  (15.181)
Role Limitation Due to Emotional Problems (RE) 49.215  (14.077) 49.400  (12.002) 49.770  (12.984) 49.770  (12.142) 49.030  (12.348)
Mental Health (MH) 48.801  (13.404) 50.007  (10.686) 50.812  (13.626) 49.471  (12.581) 50.276  (13.084)
Physical Component Score (PCS) 39.963  (11.722) 40.480  (11.113) 38.287  (12.037) 40.716  (10.711) 37.890  (12.785)
Mental Component Score (MCS) 51.510  (14.484) 53.011  (12.441) 53.120  (13.910) 51.990  (12.726) 53.389  (15.119)
14.Secondary Outcome
Title Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits'
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with data available during each window period
Arm/Group Title Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 65 47 21
Measure Type: Number
Unit of Measure: Participants
0 ER visits 84 49 41 19
1 ER visits 16 14 5 2
2 ER visits 5 2 1 0
3 ER visits 1 0 0 0
≥4 ER visits 0 0 0 0
15.Secondary Outcome
Title Healthcare Resource Utilization (HCRU) 'Mean Number of Emergency Room (ER) Visits'
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with data available during each window period
Arm/Group Title Year Prior to Baseline Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 126 106 65 47 21
Mean (Standard Deviation)
Unit of Measure: ER visits per time period
0.85  (1.492) 0.274  (0.594) 0.28  (0.516) 0.15  (0.416) 0.10  (0.301)
16.Secondary Outcome
Title Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations'
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with data available during each window period
Arm/Group Title Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 65 47 21
Measure Type: Number
Unit of Measure: Participants
0 Hospitalizations 86 58 45 20
1 Hospitalizations 17 6 1 1
2 Hospitalizations 2 1 1 0
3 Hospitalizations 1 0 0 0
≥4 Hospitalizations 0 0 0 0
17.Secondary Outcome
Title Healthcare Resource Utilization (HCRU) 'Mean Length of Stay (LOS) in Hospital'
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with data available during each window period
Arm/Group Title Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 106 65 47 21
Mean (Standard Deviation)
Unit of Measure: Days
0.8585  (2.29467) 0.6462  (2.68346) 0.25532  (1.374742) 1.1905  (5.45545)
18.Secondary Outcome
Title Healthcare Resource Utilization (HCRU) 'Number of Participants Taking Antibiotics'
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with data available during each window period
Arm/Group Title Year Prior to Baseline Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 127 106 65 47 21
Measure Type: Number
Unit of Measure: Participants
90 58 37 25 8
19.Secondary Outcome
Title Healthcare Resource Utilization (HCRU) 'Number of Antibiotic Courses'
Hide Description 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)
Time Frame One year prior to baseline to 24 months post-baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with data available during each window period
Arm/Group Title Year Prior to Baseline Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 126 106 65 47 21
Mean (Standard Deviation)
Unit of Measure: Antibiotic courses
All antibiotic courses (oral and I.V.) 2.3095  (2.84384) 0.9906  (1.27612) 0.9231  (1.07975) 0.9787  (1.40634) 0.7143  (1.18924)
Oral antibiotic courses 1.8571  (2.39571) 0.8679  (1.08738) 0.8154  (0.96651) 0.8936  (1.25515) 0.6667  (1.19722)
I.V. antibiotic courses 0.4603  (1.08555) 0.1038  (0.36330) 0.1077  (0.35895) 0.0851  (0.28206) 0.0476  (0.21822)
20.Secondary Outcome
Title Healthcare Resource Utilization (HCRU) 'Number of Participants Receiving Steroid Pulse Courses'
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with data available during each window period
Arm/Group Title Year Prior to Baseline Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 127 106 65 47 21
Measure Type: Number
Unit of Measure: Participants
65 30 14 12 4
21.Secondary Outcome
Title Healthcare Resource Utilization (HCRU) 'Number of Steroid Pulse Courses'
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with data available during each window period
Arm/Group Title Year Prior to Baseline Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 126 106 65 47 21
Mean (Standard Deviation)
Unit of Measure: Steroid Pulse Courses
All steroid pulse courses (oral and I.V.) 1.4365  (2.51554) 0.4340  (0.89463) 0.4154  (1.07372) 0.3404  (0.66844) 0.2857  (0.71714)
Oral steroid pulse courses 1.0873  (2.13174) 0.3585  (0.84164) 0.3692  (1.03937) 0.2766  (0.53981) 0.2381  (0.70034)
I.V. steroid pulse courses 0.3492  (0.96596) 0.0849  (0.28007) 0.0462  (0.21145) 0.0638  (0.24709) 0.0476  (0.21822)
22.Secondary Outcome
Title Hepatic Chemistry Parameters: Change From Baseline/Screening
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects who participated in the blood draws with data available during each window period
Arm/Group Title Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 52 34 26 15
Median (Full Range)
Unit of Measure: U/L
Alkaline phosphatase
-1.00
(-39.0 to 33.0)
1.50
(-68.0 to 35.0)
-5.50
(-70.0 to 21.0)
0.00
(-61.0 to 13.0)
ALT
0.00
(-21.0 to 27.0)
2.50
(-19.0 to 26.0)
2.00
(-21.0 to 35.0)
2.00
(-27.0 to 11.0)
AST
1.00
(-25.0 to 22.0)
0.00
(-23.0 to 9.0)
1.00
(-17.0 to 27.0)
0.00
(-10.0 to 7.0)
23.Secondary Outcome
Title Renal and Hepatic Chemistry Parameters: Change From Baseline/Screening
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects who participated in the blood draws with data available during each window period
Arm/Group Title Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 52 34 26 15
Median (Full Range)
Unit of Measure: mg/dL
Creatinine
0.02
(-0.2 to 0.6)
0.01
(-0.1 to 0.3)
0.04
(-0.2 to 0.2)
0.00
(-0.2 to 0.2)
Total bilirubin
0.00
(-0.7 to 0.6)
0.00
(-0.5 to 0.5)
0.00
(-0.3 to 0.6)
0.00
(-0.5 to 0.5)
BUN
0.65
(-7.7 to 9.8)
-0.15
(-9.2 to 10.7)
1.50
(-8.4 to 7.7)
-0.50
(-4.3 to 9.0)
24.Secondary Outcome
Title ARALAST Antibody Titers: Participants With at Least 2-Dilution Step Increases From Screening
Hide Description

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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects who participated in the blood draws with data available during each window period
Arm/Group Title Baseline to ≤6 Months >6 Months to ≤12 Months >12 Months to ≤18 Months >18 Months to ≤24 Months
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 52 34 26 15
Measure Type: Number
Unit of Measure: Participants
IgG 2 0 0 0
IgM 2 1 1 0
Time Frame Throughout the entire study period (2 years, 7 months)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Overall Study
Hide Arm/Group Description [Not Specified]
All-Cause Mortality
Overall Study
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Overall Study
Affected / at Risk (%) # Events
Total   39/127 (30.71%)    
Cardiac disorders   
Bradycardia  1/127 (0.79%)  1
Cardiac failure congestive  2/127 (1.57%)  2
Cardio-respiratory arrest  1/127 (0.79%)  1
Myocardial infarction  1/127 (0.79%)  1
Gastrointestinal disorders   
Colon gangrene  1/127 (0.79%)  1
Gastric ulcer  1/127 (0.79%)  1
Vomiting  1/127 (0.79%)  1
General disorders   
Chest pain  2/127 (1.57%)  2
Fatigue  1/127 (0.79%)  1
Hernia  2/127 (1.57%)  2
Hepatobiliary disorders   
Hepatic failure  1/127 (0.79%)  1
Infections and infestations   
Gastroenteritis  1/127 (0.79%)  1
Pneumonia  6/127 (4.72%)  7
Pseudomonas infection  1/127 (0.79%)  2
Respiratory tract infection  3/127 (2.36%)  3
Injury, poisoning and procedural complications   
Road traffic accident  2/127 (1.57%)  2
Musculoskeletal and connective tissue disorders   
Intervertebral disc protrusion  1/127 (0.79%)  1
Muscular weakness  1/127 (0.79%)  1
Nervous system disorders   
Disorientation  2/127 (1.57%)  2
Respiratory, thoracic and mediastinal disorders   
Chronic obstructive pulmonary disease  11/127 (8.66%)  13
Respiratory failure  5/127 (3.94%)  5
Vascular disorders   
Peripheral artery aneurysm  1/127 (0.79%)  1
1
Term from vocabulary, MedDRA (Unspecified)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Overall Study
Affected / at Risk (%) # Events
Total   97/127 (76.38%)    
Gastrointestinal disorders   
Diarrhea  7/127 (5.51%)  21
General disorders   
Fatigue  15/127 (11.81%)  123
Infusion related reaction  9/127 (7.09%)  13
Immune system disorders   
Hypersensitivity  13/127 (10.24%)  28
Infections and infestations   
Influenza  7/127 (5.51%)  27
Lower respiratory tract infection  24/127 (18.90%)  35
Oral fungal infection  10/127 (7.87%)  18
Respiratory tract infection  33/127 (25.98%)  66
Upper respiratory tract infection  28/127 (22.05%)  45
Nervous system disorders   
Headache  23/127 (18.11%)  98
Respiratory, thoracic and mediastinal disorders   
Chronic obstructive pulmonary disease  23/127 (18.11%)  33
Surgical and medical procedures   
Elective procedure  8/127 (6.30%)  9
Vascular disorders   
Poor venous access  8/127 (6.30%)  10
1
Term from vocabulary, MedDRA (Unspecified)
Study was terminated early due to Aralast being phased out of the market.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Name/Title: David Gelmont, MD Global Medical Director, Head of Specialty Products TA, BioTherapeutics
Organization: Baxter Healthcare Corporation
Responsible Party: Shire ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT00313144     History of Changes
Other Study ID Numbers: 450501
First Submitted: April 10, 2006
First Posted: April 12, 2006
Results First Submitted: March 4, 2011
Results First Posted: August 26, 2011
Last Update Posted: February 12, 2018