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

Leber Hereditary Optic Neuropathy Clinical Trial

— rAAV2-ND4  

Official title:

Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01267422
• Other study ID #: RAVCT-2
• Status: Completed
• Phase: N/A
• First received: December 27, 2010
• Last updated: January 7, 2018
• Start date: April 2011
• Est. completion date: November 2015

Study information

• Verified date: January 2018
• Source: Huazhong University of Science and Technology
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: November 2015
• Est. primary completion date: November 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 60 Years

Eligibility

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.

Related Conditions & MeSH terms

• Leber Hereditary Optic Neuropathy
• Optic Atrophy, Hereditary, Leber
• Optic Nerve Diseases
• Peripheral Nervous System Diseases

Intervention

Drug:
• rAAV2-ND4
injection

Locations

Country	Name	City	State
China	Department of Ophthalmology ,Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology	Wuhan	Hubei

Sponsors (2)

Lead Sponsor	Collaborator
Bin Li	Huazhong University of Science and Technology

Country where clinical trial is conducted

China, 

References & Publications (31)

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* Note: There are 31 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Best Corrected Visual Acuity(BCVA)		Up to 3 years
Primary	Results of CD3/CD4/CD8 Test	The mean percentage of CD3+/CD4+/CD8+ test before and after treatment	up to 6 months
Secondary	Intraocular Pressure;		Up to 3 years
Secondary	Neutralizing Antibody Assay	The mean of Neutralizing antibody assay of 8 patients before and after treatment	up to 3 years
Secondary	Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test	Average RNFL thickness of 8 patients througth Optical coherence tomography(OCT) test before and after treatment	Up to 3 years
Secondary	Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)	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
Secondary	Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)	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