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Gene Therapy Trial Report

Summary

Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy


NCTID NCT05858983 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Biallelic RPE65 Mutation-associated Retinal Dystrophy
Disease Ontology Term DOID:0110016; DOID:0110353
Compound Name FT-001
Compound Description AAV2-hRPE65
Sponsor Frontera Therapeutics
Funder Type Industry
Recruitment Status
Enrollment Count 9 (ESTIMATED)
Results Posted Not Available

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
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

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
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Subjects who are able to understand and sign the ICF 2. Female or male aged 8-45 years old when signing the ICF 3. Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy Exclusion Criteria: 1. Other interfering eye diseases 2. Presence of any systemic or ocular disease that can cause or likely to cause vision loss 3. There is evidence of obviously uncontrolled concomitant diseases 4. Known to have active or suspected autoimmune diseases 5. With active systemic infection under treatment 6. Pregnant or lactating women 7. Other conditions unsuitable for the study as determined by the investigator
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links