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Trial record 1 of 25 for:    "Leber hereditary optic neuropathy" OR "Hereditary Optic Atrophy"
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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 : May 2, 2016
Information provided by (Responsible Party):

Study Description
Brief Summary:
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.

Condition or disease Intervention/treatment
Leber Hereditary Optic Neuropathy Drug: rAAV2-ND4

Detailed Description:

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.

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy
Study Start Date : April 2011
Primary Completion Date : November 2015
Study Completion Date : November 2015

Arms and Interventions

Arm Intervention/treatment
Experimental: rAAV2-ND4
Drug: rAAV2-ND4
Other Name: rAAV2-ND4 gene therapy

Outcome Measures

Primary Outcome Measures :
  1. The Best Corrected Visual Acuity(BCVA) [ Time Frame: Up to 3 years ]
  2. Results of CD3/CD4/CD8 Test [ Time Frame: up to 6 months ]
    The mean percentage of CD3+/CD4+/CD8+ test before and after treatment

Secondary Outcome Measures :
  1. Intraocular Pressure; [ Time Frame: Up to 3 years ]
  2. Neutralizing Antibody Assay [ Time Frame: up to 3 years ]
    The mean of Neutralizing antibody assay of 8 patients before and after treatment

  3. 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

  4. 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.

  5. 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.

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01267422

China, Hubei
Department of Ophthalmology ,Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wu Han, Hubei, China, 430030
Sponsors and Collaborators
Bin Li
Huazhong University of Science and Technology
Study Chair: bin Li, PhD,MD Deputy Director of Ophthalmology
More Information


Responsible Party: Bin Li, Safety and Efficacy Study of rAAV2-ND4 Treatment of LHON, Huazhong University of Science and Technology
ClinicalTrials.gov Identifier: NCT01267422     History of Changes
Other Study ID Numbers: RAVCT-2
First Posted: December 28, 2010    Key Record Dates
Results First Posted: May 2, 2016
Last Update Posted: May 2, 2016
Last Verified: March 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Bin Li, Huazhong University of Science and Technology:
Leber's Hereditary Optic Neuropathy,11778 LHON mutation,
rAAV2-ND4,triamcinolone acetonide

Additional relevant MeSH terms:
Peripheral Nervous System Diseases
Optic Nerve Diseases
Optic Atrophy, Hereditary, Leber
Neuromuscular Diseases
Nervous System Diseases
Cranial Nerve Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases