ARALAST alpha1-proteinase Inhibitor (α1-PI) Surveillance Study
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 发起方
- 入组人数
- 127
- 试验地点
- 2
- 主要终点
- HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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)
结局指标
主要结局
HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months
时间窗: 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.
HRQoL 'Role Limitation Due to Physical Health (RP)' From Baseline to ≤6 Months
时间窗: 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.
HRQoL 'Bodily Pain (BP)' From Baseline to ≤6 Months
时间窗: 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.
HRQoL 'General Health (GH)' From Baseline to ≤6 Months
时间窗: 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.
HRQoL 'Vitality (VT)' From Baseline to ≤6 Months
时间窗: 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.
HRQoL 'Social Functioning (SF)' From Baseline to ≤6 Months
时间窗: 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.
HRQoL 'Role Limitation Due to Emotional Problems (RE)' From Baseline to ≤6 Months
时间窗: 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.
HRQoL 'Mental Health (MH)' From Baseline to ≤6 Months
时间窗: 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.
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
时间窗: 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.
HRQoL 'Physical Component Score (PCS)' From Baseline to ≤6 Months
时间窗: 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.
HRQoL 'Mental Component Score (MCS)' From Baseline to ≤6 Months
时间窗: 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.
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months
时间窗: 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.
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
时间窗: 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.
次要结局
- Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits'(Baseline to 24 Months)
- Healthcare Resource Utilization (HCRU) 'Mean Number of Emergency Room (ER) Visits'(One year prior to baseline to 24 months post-baseline)
- Healthcare Resource Utilization (HCRU) 'Mean Length of Stay (LOS) in Hospital'(Baseline to 24 months)
- Healthcare Resource Utilization (HCRU) 'Number of Participants Receiving Steroid Pulse Courses'(One year prior to baseline to 24 months post-baseline)
- Healthcare Resource Utilization (HCRU) 'Number of Steroid Pulse Courses'(One year prior to baseline to 24 months post-baseline)
- Hepatic Chemistry Parameters: Change From Baseline/Screening(Baseline to 24 months)
- Renal and Hepatic Chemistry Parameters: Change From Baseline/Screening(Baseline to 24 months)
- ARALAST Antibody Titers: Participants With at Least 2-Dilution Step Increases From Screening(Baseline to 24 Months)
- Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations'(Baseline to 24 Months)
- Healthcare Resource Utilization (HCRU) 'Number of Participants Taking Antibiotics'(One year prior to baseline to 24 months post-baseline)
- Healthcare Resource Utilization (HCRU) 'Number of Antibiotic Courses'(One year prior to baseline to 24 months post-baseline)