Aralast alpha1-proteinase Inhibitor Surveillance Study - Study Results

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Alpha 1-Antitrypsin (AAT) Deficiency
Intervention:	Drug: ARALAST Alpha1-Proteinase Inhibitor

Participant Flow

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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

Reporting Groups

	Description
Overall Study	Participants were treated with ARALAST according to dose and frequency of infusions recommended by their physician.

Participant Flow: Overall Study

	Overall Study
STARTED	127
COMPLETED	17
NOT COMPLETED	110
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

Baseline Characteristics

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Reporting Groups

	Description
Overall Study	No text entered.

Baseline Measures

	Overall Study
Number of Participants [units: participants]	127
Age [units: years] Median ( Full Range )	52.3 ( 21.2 to 84.8 )
Gender [units: participants]
Female	68
Male	59
Region of Enrollment [units: participants]
United States	127
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) ^[1] [units: Scores on a scale] Mean ± Standard Deviation
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]	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]

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.

Outcome Measures

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1. Primary:

HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

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2. Primary:

HRQoL 'Role Limitation Due to Physical Health (RP)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

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3. Primary:

HRQoL 'Bodily Pain (BP)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

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4. Primary:

HRQoL 'General Health (GH)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

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5. Primary:

HRQoL 'Vitality (VT)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

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6. Primary:

HRQoL 'Social Functioning (SF)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

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7. Primary:

HRQoL 'Role Limitation Due to Emotional Problems (RE)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

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8. Primary:

HRQoL 'Mental Health (MH)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

Show Outcome Measure 8

9. Primary:

HRQoL 'Physical Component Score (PCS)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

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10. Primary:

HRQoL 'Mental Component Score (MCS)' From Baseline to ≤6 Months [ Time Frame: Screening to ≤ 6 Months ]

Show Outcome Measure 10

11. 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 [ Time Frame: Baseline to 12 months ]

Show Outcome Measure 11

12. 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 [ Time Frame: Baseline to 12 months ]

Show Outcome Measure 12

13. 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 [ Time Frame: Baseline to 24 months ]

Show Outcome Measure 13

14. Secondary:

Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits' [ Time Frame: Baseline to 24 Months ]

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15. Secondary:

Healthcare Resource Utilization (HCRU) 'Mean Number of Emergency Room (ER) Visits' [ Time Frame: One year prior to baseline to 24 months post-baseline ]

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16. Secondary:

Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations' [ Time Frame: Baseline to 24 Months ]

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17. Secondary:

Healthcare Resource Utilization (HCRU) 'Mean Length of Stay (LOS) in Hospital' [ Time Frame: Baseline to 24 months ]

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18. Secondary:

Healthcare Resource Utilization (HCRU) 'Number of Participants Taking Antibiotics' [ Time Frame: One year prior to baseline to 24 months post-baseline ]

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19. Secondary:

Healthcare Resource Utilization (HCRU) 'Number of Antibiotic Courses' [ Time Frame: One year prior to baseline to 24 months post-baseline ]

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20. Secondary:

Healthcare Resource Utilization (HCRU) 'Number of Participants Receiving Steroid Pulse Courses' [ Time Frame: One year prior to baseline to 24 months post-baseline ]

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21. Secondary:

Healthcare Resource Utilization (HCRU) 'Number of Steroid Pulse Courses' [ Time Frame: One year prior to baseline to 24 months post-baseline ]

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22. Secondary:

Hepatic Chemistry Parameters: Change From Baseline/Screening [ Time Frame: Baseline to 24 months ]

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23. Secondary:

Renal and Hepatic Chemistry Parameters: Change From Baseline/Screening [ Time Frame: Baseline to 24 months ]

Show Outcome Measure 23

24. Secondary:

ARALAST Antibody Titers: Participants With at Least 2-Dilution Step Increases From Screening [ Time Frame: Baseline to 24 Months ]

Show Outcome Measure 24

Serious Adverse Events

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Other Adverse Events

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More Information

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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.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: 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.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Study was terminated early due to Aralast being phased out of the market.

Results Point of Contact:

Name/Title: David Gelmont, MD Global Medical Director, Head of Specialty Products TA, BioTherapeutics
Organization: Baxter Healthcare Corporation
e-mail: david_gelmont@baxter.com

No publications provided

Responsible Party:	Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:	NCT00313144 History of Changes
Other Study ID Numbers:	450501
Study First Received:	April 10, 2006
Results First Received:	March 4, 2011
Last Updated:	August 23, 2011
Health Authority:	United States: Food and Drug Administration