Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations

NCTID	NCT05293626 (View at clinicaltrials.gov)
Description	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 ≥ 18 years old and ≤ 75 years old to receive a single unilateral intravitreal (IVT) injection of NR082 to evaluate its 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 (a CLIA-certified laboratory) and reduced visual acuity lasted for \> 6 months and \< 10 years. (Show More)
Development Status	Active
Indication	Leber Hereditary Optic Neuropathy (LHON)
Disease Ontology Term	DOID:705
Compound Name	OPVIKA
Compound Alias	NFS-01
Sponsor	Neurophth Therapeutics Inc
Funder Type	Other
Recruitment Status	Active not recruiting
Enrollment Count	12
Results Posted	Not Available

Target Gene/Variant	ND4G11778A
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravitreal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type	none
Dose 1	Dose de-escalation: 0.5E9, 0.05mL
Dose 2	Starting dose: 1.5E9 vg, 0.05ml/eye
Dose 3	Intermediate dose: 3.0E9, 0.05mL
Dose 4	High dose: 4.5E9, 0.05mL
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	Granted ODD by FDA and EMA, completed US enrollment February 2024