Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)

This study has been completed.

Study NCT00396006 Information provided by Baxter Healthcare Corporation

First Received on November 3, 2006. Last Updated on March 3, 2011 History of Changes

Results First Received: December 15, 2010

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 Deficiency
Intervention:	Biological: Alpha1-Proteinase Inhibitor

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Participants were recruited at 5 hospital sites in New Zealand and Australia. The period studied was 1 year and 2 months.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
21 participants enrolled: 4 were screen failures (i.e., did not meet inclusion/exclusion criteria), 3 were discontinued due to unevaluable baseline bronchoalveolar lavage (BAL) samples, and 1 subject withdrew consent prior to receiving investigational product.

Reporting Groups

	Description
Participants Treated With ARALAST Fraction IV-1 (Fr. IV-1)	Weekly infusions of ARALAST Fr. IV-1 were administered to participants at a dosage of 60 mg/kg

Participant Flow: Overall Study

	Participants Treated With ARALAST Fraction IV-1 (Fr. IV-1)
STARTED	13
COMPLETED	13
NOT COMPLETED	0

Baseline Characteristics

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Reporting Groups

	Description
Participants Treated With ARALAST Fr. IV-1	No text entered.

Baseline Measures

	Participants Treated With ARALAST Fr. IV-1
Number of Participants [units: participants]	13
Age [units: participants]
<=18 years	0
Between 18 and 65 years	10
>=65 years	3
Age [units: years] Median ( Full Range )	59.6 ( 36.0 to 76.1 )
Gender [units: participants]
Female	6
Male	7
Region of Enrollment [units: participants]
Australia	8
New Zealand	5

Outcome Measures

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1. Primary:

Change in Bronchoalveolar Lavage (BAL) Epithelial Lining Fluid (ELF) Alpha1-Proteinase Inhibitor (α1-PI) Level [ Time Frame: BAL procedures were performed at baseline and after 8 consecutive weeks of treatment (minimum of 12 weeks) ]

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2. Primary:

The Number of Adverse Events (AEs) Related to the Infusion of ARALAST Fr. IV 1 Administered at a Rate of 0.2 mL/kg/Min [ Time Frame: During 8 consecutive weeks of treatment ]

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3. Primary:

Number of Changes in the Rate of Infusion [ Time Frame: During 8 consecutive weeks of treatment ]

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4. Secondary:

Ratio of Post- to Pre-treatment BAL ELF Antineutrophil Elastase Capacity (ANEC) Levels [ Time Frame: BAL procedures were performed at baseline and after 8 consecutive weeks of treatment (minimum of 12 weeks) ]

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5. Secondary:

Change in in the Ratio of BAL ELF α1-PI to Human Neutrophil Elastase (HNE) Complex Concentration [ Time Frame: BAL procedures were performed at baseline and after 8 consecutive weeks of treatment (minimum of 12 weeks) ]

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6. Secondary:

Change in the α1-PI Plasma Level [ Time Frame: Blood samples were collected at baseline and after 8 consecutive weeks of treatment ]

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7. Secondary:

Change in the Plasma Antineutrophil Elastase Capacity (ANEC) Level [ Time Frame: Blood samples were collected at baseline and after 8 consecutive weeks of treatment ]

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8. Secondary:

Clinically Significant Changes in Vital Signs From Pre- to Post-Infusion [ Time Frame: During 8 consecutive weeks of infusion ]

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9. Other Pre-specified:

Change in the BAL ELF Free Neutrophil Elastase Level [ Time Frame: BAL procedures were performed at baseline and after 8 consecutive weeks of treatment (minimum of 12 weeks) ]

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10. Other Pre-specified:

Ratio of Post- to Pre-treatment BAL ELF Total Neutrophil Elastase Level [ Time Frame: BAL procedures were performed at baseline and after 8 consecutive weeks of treatment (minimum of 12 weeks) ]

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11. Other Pre-specified:

Ratio of Post- to Pre-treatment BAL ELF Interleukin 8 (IL-8) Level [ Time Frame: BAL procedures were performed at baseline and after 8 consecutive weeks of treatment (minimum of 12 weeks) ]

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12. Other Pre-specified:

Change in the BAL ELF Tumor Necrosis Factor-alpha (TNF-α) From Baseline to Post-treatment [ Time Frame: BAL procedures were performed at baseline and after 8 consecutive weeks of treatment (minimum of 12 weeks) ]

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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: The data from the study shall be published (if applicable) by the sponsor. The investigators and the steering committee may be involved if agreed to by the sponsor. Neither the investigator nor any other person within his/her institution has the right to publish separately or individually without permission by the sponsor.

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
Isoelectric focusing (IEF) analyses that were intended to compare the profile of plasma α1-PI & ELF α1-PI were not carried out due to fact that ELF samples were either missing, insufficient or too dilute for such assays.

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:	David Gelmont, MD Global Medical Director, Head of Specialty Products TA, BioTherapeutics, Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:	NCT00396006 History of Changes
Other Study ID Numbers:	460502
Study First Received:	November 3, 2006
Results First Received:	December 15, 2010
Last Updated:	March 3, 2011
Health Authority:	United States: Food and Drug Administration