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Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Applied Genetic Technologies Corp
ClinicalTrials.gov Identifier:
NCT01054339
First received: January 21, 2010
Last updated: March 6, 2013
Last verified: March 2013
History of Changes
Results First Received: August 14, 2012  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Alpha-1 Antitrypsin Deficiency
Intervention: Drug: rAAV1-CB-hAAT

  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
Subjects were recruited from two clinical trial sites (University of Massachusetts Medical Center and Cincinatti Children's Hospital Medical Center).

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Low Dose rAAV1-CB-hAAT at dosage level of 6 x 10e11 vg/kg administered as 10 IM injections in one arm
Middle Dose rAAV1-CB-hAAT at dosage level of 1.9 x 10e12 vg/kg administered as 10 IM injections in one arm and 11 IM injections in each leg (total of 32 injections)
High Dose rAAV1-CB-hAAT at dosage level of 6 x 10e12 vg/kg administered as 10 IM injections in each arm and 10 IM injections in each of four sites in each leg (total of 100 injections)

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



  Baseline Characteristics
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Reporting Groups
  Description
Low Dose rAAV1-CB-hAAT at dosage level of 6 x 10e11 vg/kg administered as 10 IM injections in one arm
Middle Dose rAAV1-CB-hAAT at dosage level of 1.9 x 10e12 vg/kg administered as 10 IM injections in one arm and 11 IM injections in each leg (total of 32 injections)
High Dose rAAV1-CB-hAAT at dosage level of 6 x 10e12 vg/kg administered as 10 IM injections in each arm and 10 IM injections in each of four sites in each leg (total of 100 injections)
Total Total of all reporting groups

Baseline Measures
    Low Dose     Middle Dose     High Dose     Total  
Number of Participants  
[units: participants]
 		  3     3     3     9  
Age  
[units: participants]
 		       
<=18 years     0     0     0     0  
Between 18 and 65 years     2     3     3     8  
>=65 years     1     0     0     1  
Age  
[units: years]
Mean ± Standard Deviation 		  50.3  ± 26.4     48.7  ± 9.3     54.3  ± 3.5     51.1  ± 14.3  
Gender  
[units: participants]
 		       
Female     3     2     2     7  
Male     0     1     1     2  
Alpha-1 antitrypsin phenotype [1]
[units: participants]
 		       
ZZ     2     3     3     8  
SZ     1     0     0     1  
Serum total alpha-1 antitrypsin concentration [2]
[units: microMolar]
Mean ± Standard Deviation 		  6.56  ± 2.00     3.54  ± 0.12     3.45  ± 0.19     4.31  ± 1.88  
[1] Alpha-1 antitrypsin phenotype determined by isoelectric focusing gel electrophoresis
[2] Average of values at screening and baseline visits. Includes 1 phenotype SZ subject in low dose group.



  Outcome Measures
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1.  Primary:   Frequency of Grade 3 or 4 Adverse Events   [ Time Frame: During 1 year after study agent administration ]
  Show Outcome Measure 1

2.  Secondary:   Changes in Serum M-specific Alpha-1 Antitrypsin Concentration   [ Time Frame: During months 6-12 after study agent adminsitration ]
  Show Outcome Measure 2

3.  Secondary:   Changes in Serum Total Alpha-1 Antitrypsin Concentrations   [ Time Frame: During months 6-12 after study agent adminstration ]
  Show Outcome Measure 3


  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:
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.


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.  


Results Point of Contact:  
Name/Title: Jeffrey D. Chulay, MD
Organization: Applied Genetic Technologies Corp.
phone: 386-462-2204 ext 205
e-mail: jchulay@agtc.com


Publications of Results:

Other Publications:


Responsible Party: Applied Genetic Technologies Corp
ClinicalTrials.gov Identifier: NCT01054339     History of Changes
Other Study ID Numbers: AGTC-AAT-002, 2009‐014286‐20, R01HL069877, R01 FD003896
Study First Received: January 21, 2010
Results First Received: August 14, 2012
Last Updated: March 6, 2013
Health Authority: United States: Food and Drug Administration
Ireland: Irish Medicines Board