NCT03153293

A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy

• Status: Unknown
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 159 (actual)
• Sponsor: Huazhong University of Science and Technology · Academic / Other
• Sex: All
• Age: 10 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Leber Hereditary Optic Neuropathy

Interventions

Type	Name	Description
DRUG	rAAV2-ND4	a Single IVT Injection

Timeline

• Start date: 2017-12-27
• Primary completion: 2020-01-15
• Completion: 2025-01-15
• First posted: 2017-05-15
• Last updated: 2020-09-17

Locations

1 site across 1 country: China