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

NCT01267422 | Completed Results DMC

• 1 other identifier: RAVCT-2
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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.

Conditions

Leber Hereditary Optic Neuropathy

Keywords

Leber's Hereditary Optic Neuropathy,11778 LHON mutation,; rAAV2-ND4,triamcinolone acetonide

Outcome Measures

Primary Outcomes (2)

• The Best Corrected Visual Acuity(BCVA)

  Up to 3 years

• Results of CD3/CD4/CD8 Test

  The mean percentage of CD3+/CD4+/CD8+ test before and after treatment

  up to 6 months

Secondary Outcomes (5)

• Intraocular Pressure;

  Up to 3 years

• Neutralizing Antibody Assay

  up to 3 years

• Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test

  Up to 3 years

• Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)

  up to 3 years

• Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)

  up to 3 years

Study Arms (1)

rAAV2-ND4

EXPERIMENTAL

injection

Drug: rAAV2-ND4

Interventions

rAAV2-ND4 DRUG

injection

Also known as: rAAV2-ND4 gene therapy

rAAV2-ND4

Eligibility Criteria

• Age: 8 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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.
• Are participating in other clinical studies of patients.
• Patients with mental disorders.

Sponsors & Collaborators

• Bin Li: lead
• Huazhong University of Science and Technology: collaborator

Study Sites (1)

Department of Ophthalmology ，Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology

Wuhan, Hubei, 430030, China

Location

Related Publications (32)

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• Wan X, Pei H, Zhao MJ, Yang S, Hu WK, He H, Ma SQ, Zhang G, Dong XY, Chen C, Wang DW, Li B. Efficacy and Safety of rAAV2-ND4 Treatment for Leber's Hereditary Optic Neuropathy. Sci Rep. 2016 Feb 19;6:21587. doi: 10.1038/srep21587.

  PMID: 26892229 RESULT

• Liu HL, Yuan JJ, Tian Z, Li X, Song L, Li B. What are the characteristics and progression of visual field defects in patients with Leber hereditary optic neuropathy: a prospective single-centre study in China. BMJ Open. 2019 Mar 15;9(3):e025307. doi: 10.1136/bmjopen-2018-025307.

  PMID: 30878986 DERIVED

MeSH Terms

Conditions

Optic Atrophy, Hereditary, Leber

Condition Hierarchy (Ancestors)

Optic Atrophies, Hereditary; Optic Atrophy; Optic Nerve Diseases; Cranial Nerve Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Eye Diseases, Hereditary; Eye Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

Early termination leading to small numbers of subjects analyzed.

Results Point of Contact

• Title: Bin Li
• Organization: Department of Ophthalmology, Tongji Hospital Tongji Medical College, Huazhong University of Science and Technology

libin-12@163.com

+86-13638673626

Study Officials

• bin Li, PhD,MD

  Deputy Director of Ophthalmology

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Safety and Efficacy Study of rAAV2-ND4 Treatment of LHON

Study Record Dates

• First Submitted: December 27, 2010
• First Posted: December 28, 2010
• Study Start: April 1, 2011
• Primary Completion: November 1, 2015
• Study Completion: November 1, 2015
• Last Updated: January 31, 2018
• Results First Posted: May 2, 2016

Record last verified: 2018-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)