ClinicalTrials.gov
ClinicalTrials.gov Menu

Aralast alpha1-proteinase Inhibitor Surveillance Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00313144
Recruitment Status : Terminated (Study terminated early due to Aralast being phased out of the market.)
First Posted : April 12, 2006
Results First Posted : August 26, 2011
Last Update Posted : February 12, 2018
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Alpha1-antitrypsin 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

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Overall Study No text entered.

Baseline Measures
   Overall Study 
Overall Participants Analyzed 
[Units: Participants]
 127 
Age 
[Units: Years]
Median (Full Range)
 52.3 
 (21.2 to 84.8) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      68  53.5% 
Male      59  46.5% 
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.


  Outcome Measures

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

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

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

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

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

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

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

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

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

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

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 ]

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 ]

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 ]

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

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 ]

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

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

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

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

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 ]

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

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

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

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


  Serious Adverse Events


  Other Adverse Events


  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.


  More Information

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:
unchecked 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.
unchecked 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.


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



Responsible Party: Shire ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT00313144     History of Changes
Other Study ID Numbers: 450501
First Submitted: April 10, 2006
First Posted: April 12, 2006
Results First Submitted: March 4, 2011
Results First Posted: August 26, 2011
Last Update Posted: February 12, 2018