Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

NCTID	NCT05820152 (View at clinicaltrials.gov)
Description	The objective of this clinical study is to evaluate the safety, tolerability and preliminary efficacy of NFS-02 in the treatment of LHON caused by mitochondrial ND1 gene mutation. This study will enroll subjects aged ≥ 18 years old and ≤ 75 years old to receive a single unilateral intravitreal (IVT) injection of NFS-02 to evaluate its safety, tolerability and preliminary efficacy. The clinical manifestations of all subjects are to be reduced visual acuity caused by LHON associated with ND1 mutation, with laboratory test showing G3460A mutation (a CLIA-certified laboratory) and reduced visual acuity lasted for \> 6 months and \< 10 years. (Show More)
Development Status	Inactive
Indication	Leber Hereditary Optic Neuropathy (LHON)
Disease Ontology Term	DOID:705
Compound Name	NFS-02
Sponsor	Neurophth Therapeutics Inc
Funder Type	Other
Recruitment Status	Terminated
Enrollment Count	11
Results Posted	Not Available

Target Gene/Variant	ND1G3460A
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	5.0E7 vg, 0.05mL/eye
Dose 2	Starting dose: 1.5E8 vg, 0.05mL/eye
Dose 3	5.0E8 vg, 0.05mL/eye
Dose 4	1.5E9 vg, 0.05mL/eye
Dose 5

No.of Trial Sites	4
Locations	United States,China

Has US IND	True
FDA Designations
Recent Updates	Study terminated due to sponsor circumstances and external reasons