Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: FT-001 Low Dose, FT-001 Mid Dose, FT-001 High Dose.
- Who it may be relevant to
- Registry conditions: Biallelic RPE65 Mutation-associated Retinal Dystrophy. Basic parameters: 8 years — 45 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Multi-center, Open-label, Dose-escalation Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of FT-001 Administered Via Subretinal Injection in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Overview
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.
Detailed description
This study is a multi-center, open-label, phase I/II clinical study to evaluate the safety, tolerability, efficacy, immunogenicity, and in vivo biodistribution characteristics of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment.
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
Primary outcome measures
- Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs) [Time frame: 52 weeks]
Secondary outcome measures (2)
- Changes in visual function from baseline [Time frame: 52 weeks]
- Changes in visual function from baseline [Time frame: 52 weeks]
Eligibility criteria
Inclusion criteria
- Subjects who are able to understand and sign the ICF
- Female or male aged 8-45 years old when signing the ICF
- Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy
Exclusion criteria
- Other interfering eye diseases
- Presence of any systemic or ocular disease that can cause or likely to cause vision loss
- There is evidence of obviously uncontrolled concomitant diseases
- Known to have active or suspected autoimmune diseases
- With active systemic infection under treatment
- Pregnant or lactating women
- Other conditions unsuitable for the study as determined by the investigator
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Peking Union Medical College Hospital — Beijing
Identifiers
NCT: NCT05858983 · FT001-C101