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

This study is currently recruiting participants.
Verified December 2010 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: April 20, 2013
Last verified: December 2010
History of Changes

December 27, 2010
April 20, 2013
April 2011
September 2013   (final data collection date for primary outcome measure)
The visual acuity [ Time Frame: 6 month ] [ Designated as safety issue: No ]
Vision testing before and after treatment
Same as current
Complete list of historical versions of study NCT01267422 on ClinicalTrials.gov Archive Site
  • 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.
  • Intraocular pressure [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]
    Intraocular pressure testing before and after treatment
  • 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
  • OCT [ Time Frame: 6 month ] [ Designated as safety issue: No ]
    OCT testing before and after treatment
  • 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
  • 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.
Not Provided
Not Provided
 
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
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.

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.
Interventional
Not Provided
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Leber Hereditary Optic Neuropathy
Drug: rAAV2-ND4
injection
Experimental: rAAV2-ND4
injection
Intervention: Drug: rAAV2-ND4
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
6
October 2013
September 2013   (final data collection date for primary outcome measure)

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.
Both
8 Years to 60 Years
No
Contact: Bin Li, PhD,MD 8613638673626 scotopsin@163.com
Contact: Han Pei, PhD 8627-83663223 libin-12@163.com
China
 
NCT01267422
RAVCT-2
Yes
Bin Li, Huazhong University of Science and Technology
Bin Li
Huazhong University of Science and Technology
Study Chair: bin Li, PhD,MD Deputy Director of Ophthalmology
Huazhong University of Science and Technology
December 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP