Aralast alpha1-proteinase Inhibitor Surveillance Study

Alpha1-antitrypsin Deficiency Clinical Trial

Official title:

ARALAST alpha1-proteinase Inhibitor (α1-PI) Surveillance Study

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00313144
• Other study ID #: 450501
• Status: Terminated
• Phase: Phase 4
• Start date: June 9, 2006
• Est. completion date: May 1, 2009

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 127
• Est. completion date: May 1, 2009
• Est. primary completion date: December 1, 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency
• Alpha1-antitrypsin Deficiency

Intervention

Drug:
• ARALAST Alpha1-Proteinase Inhibitor
Weekly ARALAST infusions for 2 years, dose and mode of administration as prescribed by the physician

Locations

Country	Name	City	State
United States	Adupa Rao, MD	San Marino	California

Sponsors (1)

Lead Sponsor	Collaborator
Baxalta now part of Shire

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
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.	Screening to = 6 Months
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.	Screening to = 6 Months
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.	Screening to = 6 Months
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.	Screening to = 6 Months
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.	Screening to = 6 Months
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.	Screening to = 6 Months
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.	Screening to = 6 Months
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.	Screening to = 6 Months
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.	Screening to = 6 Months
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.	Screening to = 6 Months
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.	Baseline to 12 months
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.	Baseline to 12 months
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.	Baseline to 24 months
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)	Baseline to 24 Months
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)	One year prior to baseline to 24 months post-baseline
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)	Baseline to 24 Months
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)	Baseline to 24 months
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)	One year prior to baseline to 24 months post-baseline
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)	One year prior to baseline to 24 months post-baseline
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)	One year prior to baseline to 24 months post-baseline
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)	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)	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)	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	Baseline to 24 Months