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

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2013 by Huazhong University of Science and Technology
Sponsor:
Collaborator:
Huazhong University of Science and Technology
Information provided by (Responsible Party):
Bin Li, Huazhong University of Science and Technology
ClinicalTrials.gov Identifier:
NCT01267422
First received: December 27, 2010
Last updated: September 2, 2013
Last verified: September 2013
  Purpose

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


Condition Intervention
Leber Hereditary Optic Neuropathy
Drug: rAAV2-ND4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Huazhong University of Science and Technology:

Primary Outcome Measures:
  • The visual acuity [ Time Frame: 6 month ] [ Designated as safety issue: No ]
    Vision testing before and after treatment


Secondary Outcome Measures:
  • Intraocular pressure [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]
    Intraocular pressure testing before and after treatment

  • Visual evoked potential(VEP) [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]
    VEP testing before and after treatment

  • computerized visual field [ Time Frame: 6 month ] [ Designated as safety issue: No ]
    computerized visual field testing before and after treatment

  • Optical coherence tomography(OCT) [ Time Frame: 6 month ] [ Designated as safety issue: No ]
    OCT testing before and after treatment

  • Electroretinogram(ERG) [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]
    ERG testing before and after treatment

  • Liver and kidney function in plasma [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]
    Liver and kidney function in plasma testing before and after treatment

  • Enzyme-linked immunosorbent assay Enzyme-linked immunosorbent assay (ELISA) [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]
    Antibodies against AAV2 capsid was analyzed by ELISA using serum samples collected before injection and different time points after the injection.


Estimated Enrollment: 6
Study Start Date: April 2011
Estimated Study Completion Date: October 2013
Estimated Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: rAAV2-ND4
injection
Drug: rAAV2-ND4
injection

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)(0.06ml/1.5×1010; The visual acuity, visual evoked potential (VEP),optical coherence tomography( OCT), computerized visual field, Electroretinography(ERG) and Liver and kidney function in plasma were compared before and after treatment at 1,3,and 6, months interval.

  Eligibility

Ages Eligible for Study:   8 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01267422

Contacts
Contact: Bin Li, PhD,MD 8613638673626 scotopsin@163.com
Contact: Han Pei, PhD 8627-83663223 libin-12@163.com

Locations
China, Hubei
Department of Ophthalmology ,Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Recruiting
Wu Han, Hubei, China, 430030
Contact: Bin Li, PhD,MD    8613638673626    scotopsin@163.com   
Sponsors and Collaborators
Bin Li
Huazhong University of Science and Technology
Investigators
Study Chair: bin Li, PhD,MD Deputy Director of Ophthalmology
  More Information

No publications provided

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
Study First Received: December 27, 2010
Last Updated: September 2, 2013
Health Authority: China: Food and Drug Administration

Keywords provided by 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

ClinicalTrials.gov processed this record on October 19, 2014