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

January 7, 2018 updated by: Bin Li

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

9

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Hubei
      • Wuhan, Hubei, China, 430030
        • Department of Ophthalmology ，Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 58 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: rAAV2-ND4
injection
Drug: rAAV2-ND4
injection
Other Names:
  • rAAV2-ND4 gene therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The Best Corrected Visual Acuity(BCVA)
Time Frame: Up to 3 years
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
up to 6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Intraocular Pressure;
Time Frame: Up to 3 years
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
up to 3 years
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
Up to 3 years
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.
up to 3 years
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.
up to 3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bin Li

Collaborators

Huazhong University of Science and Technology

Investigators

  • Study Chair: bin Li, PhD,MD, Deputy Director of Ophthalmology

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2011

Primary Completion (Actual)

November 1, 2015

Study Completion (Actual)

November 1, 2015

Study Registration Dates

First Submitted

December 27, 2010

First Submitted That Met QC Criteria

December 27, 2010

First Posted (Estimate)

December 28, 2010

Study Record Updates

Last Update Posted (Actual)

January 31, 2018

Last Update Submitted That Met QC Criteria

January 7, 2018

Last Verified

January 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RAVCT-2

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Leber Hereditary Optic Neuropathy

Clinical Trials on rAAV2-ND4

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Albania

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe