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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
NCTID
NCT05858983
(View at clinicaltrials.gov)
Description
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
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Indication
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Compound Name
FT-001 (AAV2-hRPE65)
Sponsor
Frontera Therapeutics
Funder Type
Industry
Status
Recruiting
Enrollment Count
9
Therapy Information
Target Gene/Variant
RPE65
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Subretinal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
none
Dose 1
1.5E10 vg/eye
Dose 2
7.5E10 vg/eye
Dose 3
15E10 vg/eye
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2023-04-29
Completion Date
2029-11-30
Last Update
2023-05-15
Participation Criteria
Eligible Age
8 Years - 45 Years
Standard Ages
Child, Adult
Eligible Sex
ALL
Locations
No.of Trial Sites
1
Locations
China
Regulatory Information
Has US IND
False
Recent Updates
Resources/Links
Clinical Publications
(Abstract) Phase I study evaluating the safety, tolerability and preliminary efficacy of FT-001 gene therapy for RPE65 associated retinal dystrophy - ARVO 2024
News and Press Releases
Frontera Therapeutics Initiates Phase II Clinical Trial for FT-001 in Hereditary Retinopathy Treatment