Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
Oregon Health and Science University
University of Massachusetts, Worcester
Information provided by (Responsible Party):
Applied Genetic Technologies Corp
ClinicalTrials.gov Identifier:
NCT00749957
First received: September 8, 2008
Last updated: April 7, 2016
Last verified: April 2016
Results First Received: November 30, 2015  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Leber Congenital Amaurosis
Intervention: Biological: rAAV2-CB-hRPE65

  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
Twelve subjects with DNA sequence-confirmed mutations in RPE65 were enrolled, 10 at the Casey Eye Institute and 2 at the University of Massachusetts, between July 2009 and September 2012.

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
Lower Dose of rAAV2-CB-hRPE65 Subjects at least 6 y/o administered a lower dose of the rAAV2-CB-hRPE65 vector by subretinal injection
Higher Dose of rAAV2-CB-hRPE65 Subjects at least 6 y/o administered a higher dose of the rAAV2-CB-hRPE65 vector by subretinal injection

Participant Flow:   Overall Study
    Lower Dose of rAAV2-CB-hRPE65     Higher Dose of rAAV2-CB-hRPE65  
STARTED     6     6  
COMPLETED     6     6  
NOT COMPLETED     0     0  



  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
Lower Dose of rAAV2-CB-hRPE65 Subjects at least 6 y/o treated with a lower dose of the rAAV2-CB-hRPE65 vector by subretinal injection
Higher Dose of rAAV2-CB-hRPE65 Subjects at least 6 y/o treated with a higher dose of the rAAV2-CB-hRPE65 vector by subretinal injection
Total Total of all reporting groups

Baseline Measures
    Lower Dose of rAAV2-CB-hRPE65     Higher Dose of rAAV2-CB-hRPE65     Total  
Number of Participants  
[units: participants]
  6     6     12  
Age  
[units: participants]
     
<=18 years     3     1     4  
Between 18 and 65 years     3     5     8  
>=65 years     0     0     0  
Age  
[units: years]
Median (Full Range)
  22  
  (6 to 39)  
  31  
  (6 to 37)  
  31  
  (6 to 39)  
Gender  
[units: participants]
     
Female     2     4     6  
Male     4     2     6  
Ethnicity (NIH/OMB)  
[units: participants]
     
Hispanic or Latino     2     3     5  
Not Hispanic or Latino     4     3     7  
Unknown or Not Reported     0     0     0  
Race (NIH/OMB)  
[units: participants]
     
American Indian or Alaska Native     0     0     0  
Asian     0     2     2  
Native Hawaiian or Other Pacific Islander     0     0     0  
Black or African American     2     0     2  
White     4     4     8  
More than one race     0     0     0  
Unknown or Not Reported     0     0     0  
Region of Enrollment  
[units: participants]
     
United States     6     6     12  



  Outcome Measures
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1.  Primary:   Number of Participants Experiencing Ocular or Non-ocular Adverse Events   [ Time Frame: 2 years ]

2.  Secondary:   Participants With Changes in Visual Fields   [ Time Frame: 2 years ]

3.  Secondary:   Participants With Changes in Best Corrected Visual Acuity   [ Time Frame: 2 years ]


  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
No text entered.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.


Results Point of Contact:  
Name/Title: Chief Medical Officer
Organization: Applied Genetic Technologies Corporation
phone: 386-462-2204 ext 7205
e-mail: jchulay@agtc.com


Publications:

Responsible Party: Applied Genetic Technologies Corp
ClinicalTrials.gov Identifier: NCT00749957     History of Changes
Other Study ID Numbers: AGTC-RPE65-002
R01FD003694 ( US NIH Grant/Contract Award Number )
Study First Received: September 8, 2008
Results First Received: November 30, 2015
Last Updated: April 7, 2016
Health Authority: United States: Food and Drug Administration