Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT00263887
First received: September 12, 2005
Last updated: August 5, 2014
Last verified: August 2014
Results First Received: September 24, 2009  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Alpha 1-Antitrypsin Deficiency
Interventions: Drug: Alpha1-Proteinase Inhibitor (Human)
Drug: Albumin (Human) 20%, United States Pharmacopeia (USP)

  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
Multicenter study with 3 sites in Denmark, Sweden, and the United Kingdom.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Prolastin (60 mg/kg Body Weight) No text entered.
Placebo No text entered.

Participant Flow:   Overall Study
    Prolastin (60 mg/kg Body Weight)     Placebo  
STARTED     38     39  
COMPLETED     35     32  
NOT COMPLETED     3     7  
Withdrawal by Subject                 1                 1  
Lung transplantation                 1                 2  
Too ill to attend visit                 1                 0  
Adverse Event                 0                 2  
Disease progression                 0                 1  
Lack of Efficacy                 0                 1  



  Baseline Characteristics
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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
Prolastin (60 mg/kg Body Weight) No text entered.
Placebo No text entered.
Total Total of all reporting groups

Baseline Measures
    Prolastin (60 mg/kg Body Weight)     Placebo     Total  
Number of Participants  
[units: participants]
  38     39     77  
Age  
[units: participants]
     
<=18 years     0     0     0  
Between 18 and 65 years     33     34     67  
>=65 years     5     5     10  
Age  
[units: years]
Mean ± Standard Deviation
  54.68  ± 8.29     55.26  ± 9.68     54.97  ± 9.03  
Gender  
[units: participants]
     
Female     13     23     36  
Male     25     16     41  
Region of Enrollment  
[units: participants]
     
Denmark     18     17     35  
United Kingdom     12     14     26  
Sweden     8     8     16  
Severity of COPD at baseline [1]
[units: participants]
     
None     2     2     4  
Mild     1     2     3  
Moderate     10     8     18  
Severe     19     20     39  
Very Severe     6     7     13  
[1] Severity of Chronic Obstructive pulmonary Disease (COPD) at baseline according to Global Initiative for Chronic Obstructive Lung Disease (GOLD) classes



  Outcome Measures

1.  Primary:   The Progression Rate of Emphysema Determined by Change in 15th Percentile of Lung Density Measured by Annual CT Scan of the Whole Lung   [ Time Frame: 24 or 30 months ]

2.  Secondary:   Change in Lung Density at Each Visit as Measured by Computed Tomography   [ Time Frame: 24 or 30 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

3.  Secondary:   The Frequency of Exacerbations as Determined by Patient Diary.   [ Time Frame: 24 or 30 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   Yes

4.  Secondary:   The Deterioration of the Lung Function Will be Assessed by Measurement of the Change in Forced Expiratory Volume at One Second (FEV1) and Transfer Factor of Carbon Monoxide (KCO)   [ Time Frame: 24 or 30 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   Yes

5.  Secondary:   Duration and Severity of the Exacerbations   [ Time Frame: 24 or 30 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   Yes

6.  Secondary:   Mortality   [ Time Frame: 24 or 30 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   Yes

7.  Secondary:   Quality of Life With a Disease Specific Instrument, the St. George's Respiratory Questionnaire   [ Time Frame: 24 or 30 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   Yes


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
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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:
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: Henry Li
Organization: Talecris Biotherapeutics
phone: 1-800-520-2807
e-mail: henry.li@grifols.com


Publications of Results:
Publications automatically indexed to this study:

Responsible Party: Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT00263887     History of Changes
Other Study ID Numbers: 100533
Study First Received: September 12, 2005
Results First Received: September 24, 2009
Last Updated: August 5, 2014
Health Authority: Denmark: Danish Medicines Agency
Sweden: Medical Products Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency