Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
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
1 other identifier
interventional
9
1 country
1
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Nov 2022
Longer than P75 for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 30, 2022
CompletedFirst Submitted
Initial submission to the registry
April 29, 2023
CompletedFirst Posted
Study publicly available on registry
May 15, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 30, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
November 30, 2029
ExpectedMay 15, 2023
October 1, 2022
3 years
April 29, 2023
May 11, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
Incidence of ocular and non-ocular AEs and SAEs
52 weeks
Secondary Outcomes (2)
Changes in visual function from baseline
52 weeks
Changes in visual function from baseline
52 weeks
Study Arms (3)
FT-001 Dose 1
EXPERIMENTALIntraocular administration of a single low dose of range FT-001
FT-001 Dose 2
EXPERIMENTALIntraocular administration of a single Mid dose of range FT-001
FT-001 Dose 3
EXPERIMENTALIntraocular administration of a single High dose of range FT-001
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100142, China
Study Officials
- PRINCIPAL INVESTIGATOR
Ruifang Sui
Peking Union Medical College Hospital
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 29, 2023
First Posted
May 15, 2023
Study Start
November 30, 2022
Primary Completion
November 30, 2025
Study Completion (Estimated)
November 30, 2029
Last Updated
May 15, 2023
Record last verified: 2022-10
Data Sharing
- IPD Sharing
- Will not share