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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01267422
Recruitment Status : Completed
First Posted : December 28, 2010
Results First Posted : May 2, 2016
Last Update Posted : January 31, 2018
Sponsor:
Collaborator:
Huazhong University of Science and Technology
Information provided by (Responsible Party):
Bin Li, Huazhong University of Science and Technology

December 27, 2010
December 28, 2010
May 21, 2015
May 2, 2016
January 31, 2018
April 2011
November 2015   (Final data collection date for primary outcome measure)
  • The Best Corrected Visual Acuity(BCVA) [ Time Frame: Up to 3 years ]
  • Results of CD3/CD4/CD8 Test [ Time Frame: up to 6 months ]
    The mean percentage of CD3+/CD4+/CD8+ test before and after treatment
The visual acuity [ Time Frame: 6 month ]
Vision testing before and after treatment
Complete list of historical versions of study NCT01267422 on ClinicalTrials.gov Archive Site
  • Intraocular Pressure; [ Time Frame: Up to 3 years ]
  • Neutralizing Antibody Assay [ Time Frame: up to 3 years ]
    The mean of Neutralizing antibody assay of 8 patients before and after treatment
  • Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test [ Time Frame: Up to 3 years ]
    Average RNFL thickness of 8 patients througth Optical coherence tomography(OCT) test before and after treatment
  • Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal) [ Time Frame: up to 3 years ]
    MD: mean deviation, the value Close to 0 regarded normal.VFI/MD：The bigger one was more close to the normal value.
  • Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal) [ Time Frame: up to 3 years ]
    VFI: visual field index ,the value Close to 100% regarded normal. VFI/MD：The bigger one was more close to the normal value.
  • Intraocular pressure [ Time Frame: 6 month ]
    Intraocular pressure testing before and after treatment
  • VEP [ Time Frame: 6 month ]
    VEP testing before and after treatment
  • computerized visual field [ Time Frame: 6 month ]
    computerized visual field testing before and after treatment
  • OCT [ Time Frame: 6 month ]
    OCT testing before and after treatment
  • ERG [ Time Frame: 6 month ]
    ERG testing before and after treatment
  • Liver and kidney function in plasma [ Time Frame: 6 month ]
    Liver and kidney function in plasma testing before and after treatment
  • ELISA [ Time Frame: 6 month ]
    Antibodies against AAV2 capsid was analyzed by ELISA using serum samples collected before injection and different time points after the injection.
Not Provided
Not Provided
 
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.

Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited ocular disorder associated with a mutation in mtDNA . The common manifestation is visual loss which caused by the respiratory chain enzymes complex dysfunction resulting in increased oxidative stress enzymes production.

Material and Method Seven patients with 11778 LHON mutation were randomly treated with a Single IVT Injection of recombinant Adeno-Associated Virus-NADH dehydrogenase, subunit 4 (complex I)（rAAV2-ND4)（0.05ml).The dose was 5 × 10^9 vg/0.05 mL for patients younger than 12 years old, and 1 × 10^10 vg/0.05 mL for patients older than 12 years old. The visual acuity, visual evoked potential （VEP）,optical coherence tomography（ OCT）, computerized visual field, electroretinograms(ERG), retinal nerve fiber layer(RNFL)and Liver and kidney function in plasma were compared before and after treatment at 1,3,and 6, months interval.
Interventional
Not Applicable
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Leber Hereditary Optic Neuropathy
Drug: rAAV2-ND4
injection
Other Name: rAAV2-ND4 gene therapy
Experimental: rAAV2-ND4
injection
Intervention: Drug: rAAV2-ND4

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
9
6
November 2015
November 2015   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. comply with Leber hereditary optic neuropathy diagnostic criteria.
  2. in patients with informed consent, voluntary participation.
  3. signed informed consent.
  4. 8 ≤ Age ≤ 60 years old, good health, the patient can tolerate local anesthesia surgery.
  5. to comply with doctor's instructions, can in the time of referral.

Exclusion Criteria:

  1. Cardiopulmonary and renal function in severe weakness, cancer, a variety of bleeding disorders, acute sensing disease, high fever, high fever disease, women during pregnancy, heart disease, such as post-operative recovery period.
  2. Are participating in other clinical studies of patients.
  3. Patients with mental disorders.
Sexes Eligible for Study: All
8 Years to 60 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
China
 
 
NCT01267422
RAVCT-2
Yes
Not Provided
Plan to Share IPD: No
Bin Li, Huazhong University of Science and Technology
Bin Li
Huazhong University of Science and Technology
Study Chair: bin Li, PhD,MD Deputy Director of Ophthalmology
Huazhong University of Science and Technology
January 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP