Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
An Open-label, Single-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intraocular Administration of FT-002 in Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa.
1 other identifier
interventional
18
1 country
2
Brief Summary
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for early_phase_1
Started Feb 2023
Longer than P75 for early_phase_1
2 active sites
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
February 1, 2023
CompletedFirst Submitted
Initial submission to the registry
April 29, 2023
CompletedFirst Posted
Study publicly available on registry
May 24, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 31, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2027
ExpectedMay 24, 2023
August 1, 2022
9 months
April 29, 2023
May 23, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
Incidence of AEs
Number and proportion of Adverse Events
from FT-002 administration through up to 1 years
Secondary Outcomes (2)
Change in visual function
from FT-002 administration through up to 1 years
Change in retinal structure as assessed by Optical Coherence Tomography
from FT-002 administration through up to 1 years
Study Arms (3)
Low dose FT-002
EXPERIMENTALIntraocular injection of a single low dose of FT-002
Intermediate dose FT-002
EXPERIMENTALIntraocular injection of a single Intermediate dose of FT-002
High dose FT-002
EXPERIMENTALIntraocular injection of a single High dose of FT-002
Interventions
Comparison of different dosages of FT-002
Eligibility Criteria
You may qualify if:
- Subjects that are willing and able to follow study procedures; 2.Males aged 8-45 years old at the time of signing the Informed Consent Form; 4.Subjects who are confirmed with variants of RPGR ;
You may not qualify if:
- Have other retinal degenerative diseases, such as retinal degeneration caused by other known Inherited retinal disease gene variants or previously received an gene therapy product.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Frontera Therapeuticslead
- Eye & ENT Hospital of Fudan Universitycollaborator
- Peking Union Medical College Hospitalcollaborator
- Henan Provincial People's Hospitalcollaborator
Study Sites (2)
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100142, China
Eye & ENT hospital of Fudan university
Shanghai, Shanghai Municipality, 200032, China
Study Officials
- PRINCIPAL INVESTIGATOR
Gezhi Xu
Eye & ENT Hospital of Fudan University
- PRINCIPAL INVESTIGATOR
Ruifang Sui
Peking Union Medical College Hospital
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- early 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 24, 2023
Study Start
February 1, 2023
Primary Completion
October 31, 2023
Study Completion (Estimated)
November 1, 2027
Last Updated
May 24, 2023
Record last verified: 2022-08
Data Sharing
- IPD Sharing
- Will not share
Researchers qualified can request the dataset, including de-identified individual subject data.