A Phase 2/3 interventional study of rAAV2-ND4 in Leber Hereditary Optic Neuropathy, sponsored by Huazhong University of Science and Technology. Status unknown at 1 site in China. Open to participants aged 10 Years to 65 Years. Per ClinicalTrials.gov, last updated 2020-09-17.
Sponsored by Huazhong University of Science and Technology · Phase 2/3, Interventional, and Treatment
This study is meant to evaluate the safety and efficacy of rAAV2-ND4 treatment for Leber hereditary optic neuropathy with the G11778A mutation in mitochondrial DNA.
Leber's Optic Hereditary Neuropathy (LHON) is a maternally inherited ocular disorder primarily associated with mutations in mitochondrial DNA. The disease is a common cause of blindness in both eyes of affected teenagers and young adults. There is currently no approved effective treatment for LHON.
In 2011, the first LHON gene therapy investigator-initiated study was conducted (registered in December 2010 with ClinicalTrials.gov Identifier NCT01267422) to explore the safety and efficacy of gene therapy for LHON. The gene therapy was a recombinant adeno-associated virus serotype 2 containing human mitochondrial ND4 (MT-ND4) gene (rAAV2-ND4). By 36 months of follow-up, six out of nine patients who received the rAAV2-ND4 intravitreal injection experienced clinically significant vision improvement and no adverse events were observed.
This is an open-label, single-arm, multi-center study to further evaluate the safety and efficacy of rAAV-ND4 in the treatment of LHON patients with G11778A mutation. All patients will be treated with a single intravitreal injection rAAV-ND4, with dose 1 × 10\^10 vg/0.05 mL in one of the eyes.
128 studies on the registry are indexed under Optic Nerve Diseases; 27 are open to participants now.
This study's enrollment of 159 is above the median of 40 across 84 interventional studies indexed under Optic Nerve Diseases.
Browse Optic Nerve Diseases studies →Huazhong University of Science and Technology is the lead sponsor of 241 studies on the registry; 60 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
A Single IVT Injection of recombinant Adeno-Associated Virus-NADH dehydrogenase, subunit 4 (complex I)（rAAV2-ND4)（0.05ml).The dose is 1 × 10\^10 vg/0.05 mL for test groups.
Drug: rAAV2-ND4
a Single IVT Injection
BCVA
The Best Corrected Visual Acuity
Time frame: Change from Baseline at 12 months .
Computerized Visual Field
Time frame: Change from Baseline at 12 months .
VEP
visual evoked potential
Time frame: Change from Baseline at 12 months .
RNFL
retinal nerve fiber layer
Time frame: Change from Baseline at 12 months .
Liver function in plasma
Time frame: Change from Baseline at 12 months .
kidney function in plasma
Time frame: Change from Baseline at 12 months .
Plan to share: No
This study is status unknown, as verified in Sep 2020. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Huazhong University of Science and Technology