Clinical trial NCT00313144: Aralast alpha1-proteinase Inhibitor Surveillance Study
Official title: ARALAST alpha1-proteinase Inhibitor (α1-PI) Surveillance Study
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Alpha1-antitrypsin Deficiency
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2006-04-12
Enrollment: 127
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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)

Primary outcomes:
measure: HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL 'Role Limitation Due to Physical Health (RP)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL 'Bodily Pain (BP)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL 'General Health (GH)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL 'Vitality (VT)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL 'Social Functioning (SF)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL 'Role Limitation Due to Emotional Problems (RE)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL 'Mental Health (MH)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL 'Physical Component Score (PCS)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL 'Mental Component Score (MCS)' From Baseline to ≤6 Months
timeFrame: Screening to ≤ 6 Months
measure: HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months
timeFrame: Baseline to 12 months
measure: 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
timeFrame: Baseline to 12 months
measure: 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
timeFrame: Baseline to 24 months
Secondary outcomes:
measure: Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits'
timeFrame: Baseline to 24 Months
measure: Healthcare Resource Utilization (HCRU) 'Mean Number of Emergency Room (ER) Visits'
timeFrame: One year prior to baseline to 24 months post-baseline
measure: Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations'
timeFrame: Baseline to 24 Months
measure: Healthcare Resource Utilization (HCRU) 'Mean Length of Stay (LOS) in Hospital'
timeFrame: Baseline to 24 months
measure: Healthcare Resource Utilization (HCRU) 'Number of Participants Taking Antibiotics'
timeFrame: One year prior to baseline to 24 months post-baseline
measure: Healthcare Resource Utilization (HCRU) 'Number of Antibiotic Courses'
timeFrame: One year prior to baseline to 24 months post-baseline
measure: Healthcare Resource Utilization (HCRU) 'Number of Participants Receiving Steroid Pulse Courses'
timeFrame: One year prior to baseline to 24 months post-baseline
measure: Healthcare Resource Utilization (HCRU) 'Number of Steroid Pulse Courses'
timeFrame: One year prior to baseline to 24 months post-baseline
measure: Hepatic Chemistry Parameters: Change From Baseline/Screening
timeFrame: Baseline to 24 months
measure: Renal and Hepatic Chemistry Parameters: Change From Baseline/Screening
timeFrame: Baseline to 24 months
measure: ARALAST Antibody Titers: Participants With at Least 2-Dilution Step Increases From Screening
timeFrame: Baseline to 24 Months
Other outcomes:
Locations:
city: San Marino
country: United States
facility: Adupa Rao, MD
state: California
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: