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Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON) (rAAV2-ND4)

This study has been completed.
Sponsor:
Collaborator:
Huazhong University of Science and Technology
Information provided by (Responsible Party):
Bin Li, Huazhong University of Science and Technology
ClinicalTrials.gov Identifier:
NCT01267422
First received: December 27, 2010
Last updated: March 29, 2016
Last verified: March 2016
Results First Received: May 21, 2015  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Leber Hereditary Optic Neuropathy
Intervention: Drug: rAAV2-ND4

  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
Patients diagnosed with LHON (with a verified point mutation at the 11778 nucleotide site)

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All patients were administered rAAV2-ND4 by intravitreal injection to one eye and then followed for 36 months.

Reporting Groups
  Description
Participants Receiving Treatment in Left Eye 5 participants receiving treatment in left eye
Participants Receiving Treatment in Right Eye 4 participants receiving treatment in right eye

Participant Flow for 2 periods

Period 1:   Intravitreal rAAV2-ND4 Injection
    Participants Receiving Treatment in Left Eye   Participants Receiving Treatment in Right Eye
STARTED   5   4 
COMPLETED   5   4 
NOT COMPLETED   0   0 

Period 2:   Safety and Efficacy Study
    Participants Receiving Treatment in Left Eye   Participants Receiving Treatment in Right Eye
STARTED   5   4 
COMPLETED   5   4 
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
All Study Participants Age,Gender,Ethnicity,Race,Region of Enrollment

Baseline Measures
   All Study Participants 
Overall Participants Analyzed 
[Units: Participants]
 9 
Age 
[Units: Years]
Median (Inter-Quartile Range)
 15 
 (7.65 to 30.79) 
Gender 
[Units: Participants]
 
Female   2 
Male   7 
Ethnicity (NIH/OMB) 
[Units: Participants]
 
Hispanic or Latino   0 
Not Hispanic or Latino   9 
Unknown or Not Reported   0 
Race (NIH/OMB) 
[Units: Participants]
 
American Indian or Alaska Native   0 
Asian   9 
Native Hawaiian or Other Pacific Islander   0 
Black or African American   0 
White   0 
More than one race   0 
Unknown or Not Reported   0 
Region of Enrollment 
[Units: Participants]
 
China   9 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   The Best Corrected Visual Acuity(BCVA)   [ Time Frame: Up to 3 years ]

2.  Primary:   Results of CD3/CD4/CD8 Test   [ Time Frame: up to 6 months ]

3.  Secondary:   Intraocular Pressure;   [ Time Frame: Up to 3 years ]

4.  Secondary:   Neutralizing Antibody Assay   [ Time Frame: up to 3 years ]

5.  Secondary:   Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test   [ Time Frame: Up to 3 years ]

6.  Secondary:   Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)   [ Time Frame: up to 3 years ]

7.  Secondary:   Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)   [ Time Frame: up to 3 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
Early termination leading to small numbers of subjects analyzed.


  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: Bin Li
Organization: Department of Ophthalmology, Tongji Hospital Tongji Medical College, Huazhong University of Science and Technology
phone: +86-13638673626
e-mail: libin-12@163.com


Publications of Results:
Other Publications:


Responsible Party: Bin Li, Huazhong University of Science and Technology
ClinicalTrials.gov Identifier: NCT01267422     History of Changes
Other Study ID Numbers: RAVCT-2
Study First Received: December 27, 2010
Results First Received: May 21, 2015
Last Updated: March 29, 2016
Health Authority: China: Food and Drug Administration