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Clinical Trials in China / NCT05858983
Recruiting Phase 1/2

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

NCT05858983 · tracked via the Priya Life Science China tracker
Sponsor
Frontera Therapeutics
Phase
Phase 1/2
Started
2022-11-30
Last updated
2023-05-15

Condition(s) studied

Biallelic RPE65 Mutation-associated Retinal Dystrophy

Investigational drug(s) / intervention(s)

FT-001 Low DoseFT-001 Mid DoseFT-001 High Dose

FT-001 Low Dose: Comparison of different dosages of FT-001

FT-001 Mid Dose: Comparison of different dosages of FT-001

FT-001 High Dose: Comparison of different dosages of FT-001

Study summary

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.

Eligibility

Sex
ALL
Min age
8 Years
Max age
45 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs) — 52 weeks
    Incidence of ocular and non-ocular AEs and SAEs

Trial sites (1)

FacilityCityRegionStatus
Peking Union Medical College Hospital Beijing Beijing Municipality Recruiting

More Frontera Therapeutics trials in China

Gene Therapy(FT-003) for Wet AMD Recruiting
Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa Recruiting
Gene Therapy for Diabetic Macular Edema Recruiting
Gene Therapy for DME Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05858983 on ClinicalTrials.gov ↗ ← All trials in China