NCT07406854

A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation

A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Safety and Efficacy of Gene Therapy for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 95 (actual)
• Sponsor: Wuhan Neurophth Biotechnology Limited Company · Academic / Other
• Sex: All
• Age: 12 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

The objective of this clinical study is to evaluate the safety and efficacy of NR082 in the treatment of LHON caused by mitochondrial ND4 gene mutation. This study will enroll subjects aged ≥ 12 years old and ≤ 75 years old to receive a single bilateral intravitreal (IVT) injection of NR082 to evaluate safety and efficacy. The clinical manifestations of all subjects are to be reduced visual acuity caused by LHON associated with ND4 mutation, with laboratory test showing G11778A mutation and reduced visual acuity lasted for \>6 months and \<10 years.

Detailed description

Safety run-in phase: The safety run-in phase will enroll 6 evaluable subjects aged ≥ 12 years and ≤ 75 years , namely 4.5 x 109 vg, 0.05 mL eye/dose（bilaterally） and monitor the safety for at least 6 weeks. If there is no new safety concern evaluated by the SRC, the randomized, double-blind, sham-injection control study can be initiated. Second Stage: randomized, double-blind, sham-injection control study: The randomized, double-blind, sham-injection control study is to verify the efficacy and safety of NR082 in LHON caused by mitochondrial gene ND4 mutation . This part is divided into the NR082 treatment group and the control group (sham-injection group).

Conditions

• Leber Hereditary Optic Neuropathy (LHON)

Interventions

Type	Name	Description
GENETIC	rAAV2-ND4	a single bilateral intravitreal (IVT) injection of NR082
OTHER	Sham (No Treatment)	sham-injection

Timeline

• Start date: 2024-09-19
• Primary completion: 2026-05-30
• Completion: 2030-05-30
• First posted: 2026-02-12
• Last updated: 2026-02-12

Locations

1 site across 1 country: China