- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00313144
Aralast alpha1-proteinase Inhibitor Surveillance Study
ARALAST alpha1-proteinase Inhibitor (α1-PI) Surveillance Study
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 Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 4
Contacts and Locations
Study Locations
-
-
California
-
San Marino, California, United States, 91108
- Adupa Rao, MD
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Male or female 18 years of age or older
- Diagnosis of AAT deficiency associated emphysema
- Active prescription for augmentation therapy with ARALAST
- On service with Coram (a speciality pharmacy provider)
- Signed and dated informed consent
Exclusion Criteria:
- Clinically significant medical (other than COPD), psychiatric, or cognitive illness that, in the opinion of Coram or the sponsor or the investigator, may compromise subject safety or compliance (such as end stage renal or hepatic or heart disease, or metastatic cancer or any difficulty in communicating over the telephone lines)
- Previous treatment with ARALAST (i.e. subjects who had previously received and then discontinued ARALAST augmentation therapy and are now restarting ARALAST will be excluded from the study)
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months
Time Frame: Screening 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
Time Frame: Screening 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
Time Frame: Screening 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
Time Frame: Screening 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 'Vitality (VT)' From Baseline to ≤6 Months
Time Frame: Screening 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
Time Frame: Screening 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
Time Frame: Screening 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
Time Frame: Screening 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
Time Frame: Screening 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
Time Frame: Screening 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
Time Frame: Baseline 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
Time Frame: Baseline 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, >12 Months to ≤18 Months, and >18 Months to ≤24 Months
Time Frame: Baseline 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
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits'
Time Frame: Baseline to 24 Months
|
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
|
Healthcare Resource Utilization (HCRU) 'Mean Number of Emergency Room (ER) Visits'
Time Frame: One year prior to baseline to 24 months post-baseline
|
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'
Time Frame: Baseline to 24 Months
|
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'
Time Frame: Baseline to 24 months
|
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'
Time Frame: One year prior to baseline to 24 months post-baseline
|
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'
Time Frame: One year prior to baseline to 24 months post-baseline
|
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'
Time Frame: One year prior to baseline to 24 months post-baseline
|
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'
Time Frame: One year prior to baseline to 24 months post-baseline
|
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
Time Frame: Baseline to 24 months
|
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
Time Frame: Baseline to 24 months
|
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
Time Frame: Baseline to 24 Months
|
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
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Respiratory Tract Diseases
- Lung Diseases
- Liver Diseases
- Genetic Diseases, Inborn
- Subcutaneous Emphysema
- Emphysema
- Alpha 1-Antitrypsin Deficiency
- Molecular Mechanisms of Pharmacological Action
- Enzyme Inhibitors
- Serine Proteinase Inhibitors
- Trypsin Inhibitors
- Protease Inhibitors
- Alpha 1-Antitrypsin
Other Study ID Numbers
- 450501
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Alpha1-antitrypsin Deficiency
-
Baxalta now part of ShireArriva Pharmaceuticals, Inc.CompletedAlpha1-antitrypsin DeficiencyUnited States
-
Grifols Therapeutics LLCGrifols Japan K.K.CompletedAlpha1-Antitrypsin DeficiencyJapan
-
Baxalta now part of ShireArriva Pharmaceuticals, Inc.CompletedAlpha1-antitrypsin DeficiencyUnited States
-
Baxalta now part of ShireCompleted
-
RWTH Aachen UniversityUnknown
-
TakedaEnrolling by invitationAlpha1-Antitrypsin DeficiencyUnited Kingdom, Germany, United States, Austria, Portugal
-
Grifols Therapeutics LLCRecruiting
-
Vertex Pharmaceuticals IncorporatedCompletedAlpha1-Antitrypsin DeficiencyUnited States, Germany, Canada, Ireland, Sweden, United Kingdom
-
Grifols Therapeutics LLCGrifols Japan K.K.Completed
-
Baxalta now part of ShireCompletedAlpha1-antitrypsin DeficiencyUnited States, Canada
Clinical Trials on ARALAST Alpha1-Proteinase Inhibitor
-
Baxalta now part of ShireCompletedAlpha 1-Antitrypsin DeficiencyNew Zealand, Australia
-
Baxalta now part of ShireBaxalta Innovations GmbH, now part of ShireTerminatedChronic Obstructive Pulmonary Disease | Alpha1-antitrypsin DeficiencyUnited States, Canada, Australia
-
ShireWithdrawnChronic Obstructive Pulmonary Disease | Alpha1-antitrypsin Deficiency
-
TakedaWithdrawnChronic Obstructive Pulmonary Disease (COPD) | Alpha1-Antitrypsin Deficiency
-
Blessing Corporate Services, IncTakeda Pharmaceuticals North America, Inc.Withdrawn
-
Grifols Therapeutics LLCTerminatedCOVID-19United States, Brazil, Chile, Colombia, Mexico
-
Baxalta now part of ShireBaxter Healthcare, Ltd. (New Zealand), Baxter Healthcare Pty. Ltd. (Australia)CompletedAlpha 1-Antitrypsin DeficiencyAustralia, New Zealand
-
Grifols Therapeutics LLCTerminatedType 1 Diabetes MellitusUnited States
-
Grifols Therapeutics LLCCompletedEmphysema | Alpha 1-antitrypsin Deficiency (AATD)United States
-
National Institute of Allergy and Infectious Diseases...Juvenile Diabetes Research Foundation; Immune Tolerance Network (ITN)WithdrawnDiabetes Mellitus, Type 1United States