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

NCT05858983 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2023-05-15

No results posted yet for this 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.

Conditions

  • Biallelic RPE65 Mutation-associated Retinal Dystrophy

Interventions

GENETIC

FT-001 Low Dose

Comparison of different dosages of FT-001

GENETIC

FT-001 Mid Dose

Comparison of different dosages of FT-001

GENETIC

FT-001 High Dose

Comparison of different dosages of FT-001

Sponsors & Collaborators

  • Frontera Therapeutics

    lead INDUSTRY

Principal Investigators

  • Ruifang Sui · Peking Union Medical College Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
8 Years
Max Age
45 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-11-30
Primary Completion
2025-11-30
Completion
2029-11-30

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05858983 on ClinicalTrials.gov