Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

NCTID	NCT06492850 (View at clinicaltrials.gov)
Description	The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase). (Show More)
Development Status	Active
Indication	X-Linked Retinitis Pigmentosa (XLRP)
Disease Ontology Term	DOID:0110414
Compound Name	FT-002
Compound Description	AAV5-GRK1-hRPGRORF15
Sponsor	Frontera Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	32 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	RPGRORF15
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intraocular
Drug Product Type	Viral vector
Target Tissue/Cell	Photoreceptors
Delivery System	Viral transduction
Vector Type	AAV5
Editor Type	none
Dose 1	5E10 vg/eye
Dose 2	10E10 vg/eye
Dose 3	20E10 vg/eye

Eligible Age	8 Years - 45 Years
Standard Ages	Child, Adult
Sexes Eligible for Study	MALE
Eligibility Criteria	Inclusion Criteria: * Subjects that are willing and able to follow study procedures including scheduled visits, treatment plan, and laboratory tests, and sign a written informed consent form; * Age: Phase I dose escalation stage, 18-45 years old male (including boundary value) at the time of signing the ICF; Phase II dose extension stage, males 8-45 years old (including boundary values) at the time of signing the ICF; * Clinically diagnosed XLRP, the main symptoms include but are not limited to night blindness, visual field loss, vision loss, etc.; Exclusion Criteria: * Have other retinal degenerative diseases, such as retinal degeneration caused by other known Inherited retinal disease gene variants or previously received an gene therapy product. View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	1
Locations	China

Has US IND	False
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	FDA clears IND for Phase 2 clinical trials 9/23/24