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Trial record 46 of 76 for:    ALPHA-1-ANTITRYPSIN DEFICIENCY

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency (AAT)

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ClinicalTrials.gov Identifier: NCT00430768
Recruitment Status : Completed
First Posted : February 2, 2007
Results First Posted : December 15, 2016
Last Update Posted : December 15, 2016
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Applied Genetic Technologies Corp
Alpha-1 Foundation
University of Florida
National Center for Research Resources (NCRR)
Information provided by (Responsible Party):
Terence Flotte, University of Massachusetts, Worcester

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Alpha 1-Antitrypsin Deficiency
Intervention: Biological: rAAV1-CB-hAAT

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Subjects were recruited to the University of Florida, Clinical Research Center for the active study; long term follow up was completed at the University of Massachusetts, Medical School.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Subjects on alpha-1 antitrypsin (AAT) protein replacement product prior to study, discontinued treatment 4 weeks (Group 1) and 8 weeks (Groups 2 and 3) prior to study agent administration and were able to resume treatment 11 weeks after study agent had been administered. The group was determined by when the subject joined the study.

Reporting Groups
  Description
Group 1 Low Dose

6.9 x10e12 vector genomes (vg)

Group 1 receives rAAV1-CB-hAAT 6.9 x10e12 vg.

Group 2 Middle Dose

2.1 x 10e13 vector genomes; 2.2 x 10e13 vector genomes

Group 2, 1 subject received rAAV1-CB-hAAT 2.1 x10e13 vg; 202 and 203 received rAAV1-CB-hAAT 2.2 x10e13 vg

Group 3 High Dose

rAAV1-CB-hAAT 6.0 x10e13 vg

Group 3 receives rAAV1-CB-hAAT 6.0 x10e13 vg


Participant Flow:   Overall Study
    Group 1 Low Dose   Group 2 Middle Dose   Group 3 High Dose
STARTED   3   3   3 
COMPLETED   3   3   3 
NOT COMPLETED   0   0   0 



  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
Group 1 Low Dose

6.9 x10e12 vector genomes

Group 1 receives rAAV1-CB-hAAT 6.9 x1012 vg (vector genomes), e

Group 2 Middle Dose

2.1 x 10e13 vector genomes

Group 2 receives rAAV1-CB-hAAT 2.1 x1013 vg

Group 3 High Dose

rAAV1-CB-hAAT 6.0 x10e13 vg

Group 2 receives rAAV1-CB-hAAT 2.1 x1013 vg

Total Total of all reporting groups

Baseline Measures
   Group 1 Low Dose   Group 2 Middle Dose   Group 3 High Dose   Total 
Overall Participants Analyzed 
[Units: Participants]
 3   3   3   9 
Age, Customized 
[Units: Years]
Median (Full Range)
 69 
 (66 to 73) 
 54 
 (38 to 59) 
 47 
 (35 to 61) 
 54 
 (35 to 73) 
Gender 
[Units: Participants]
Count of Participants
       
Female      2  66.7%      0   0.0%      2  66.7%      4  44.4% 
Male      1  33.3%      3 100.0%      1  33.3%      5  55.6% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
       
Hispanic or Latino      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Not Hispanic or Latino      3 100.0%      3 100.0%      3 100.0%      9 100.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
       
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Black or African American      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
White      3 100.0%      3 100.0%      3 100.0%      9 100.0% 
More than one race      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Region of Enrollment 
[Units: Participants]
       
United States   3   3   3   9 
FEV1 (% predicted) [1] 
[Units: Percent predicted]
Median (Full Range)
 86.7 
 (53.6 to 92.1) 
 50.8 
 (40.5 to 93.0) 
 58.0 
 (54.9 to 97.6) 
 58.0 
 (40.5 to 97.6) 
Weight (kg) 
[Units: Kilograms]
Median (Full Range)
 66.5 
 (54.6 to 73.7) 
 83.0 
 (72.7 to 89.0) 
 72.6 
 (69.3 to 134.6) 
 72.6 
 (54.6 to 134.6) 
Prior AAT Protein Augmentation Therapy 
[Units: Participants]
       
Yes   2   1   1   4 
No   1   2   2   5 


  Outcome Measures

1.  Primary:   Adverse Events Possibly, Probably or Definitely Related to Study Drug   [ Time Frame: During 1 year after study agent administration ]

2.  Secondary:   hAAT Expression in Blood Measured Using M-specific Allele ELISA   [ Time Frame: Baseline, Days 14, 30, 45, 60, 90, (180, 270, and 365 if not on protein replacement therapy) ]


  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 results based on small number of subjects No statistical analysis.


  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 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.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Terry Flotte
Organization: UMASS Medical School
phone: (508) 856-2107
e-mail: Terry.Flotte@umassmed.edu


Publications:

Responsible Party: Terence Flotte, University of Massachusetts, Worcester
ClinicalTrials.gov Identifier: NCT00430768     History of Changes
Other Study ID Numbers: 438
5R01HL069877 ( U.S. NIH Grant/Contract )
NIH-OBA 0404-638 ( Registry Identifier: Gene Therapy Protocol Number )
NCRR-supported GCRC# 611 ( Other Identifier: University of Florida )
UF IBC RD 2630 ( Other Identifier: University of Florida )
AGTC-AAV1-001 ( Other Identifier: Applied Genetics Technologies C )
WIRB # 20052374 ( Other Identifier: Western Institutional Review B )
BB-IND 12728 ( Other Identifier: FDA )
RR00032, RR00082 ( Other Grant/Funding Number: NCRR )
First Submitted: January 31, 2007
First Posted: February 2, 2007
Results First Submitted: September 21, 2016
Results First Posted: December 15, 2016
Last Update Posted: December 15, 2016