Actively Recruiting
A Phase I/II Study to Evaluate Safety and Efficacy of FT-002 Subretinal Injection in Males With RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
Led by Frontera Therapeutics · Updated on 2024-07-09
32
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and effects of a one-time subretinal injection of FT-002 in male participants aged 8 to 45 who have X-linked retinitis pigmentosa (XLRP) caused by a mutation in the RPGR gene. This study includes both Phase I, which focuses on dose escalation, and Phase II, which focuses on dose expansion to better understand the treatment's impact. Participants will receive a single intraocular injection of FT-002 at either a low or high dose. The study is not randomized and does not use masking, meaning both participants and researchers know which dose is given. The trial is designed to first test safety and tolerability, then expand to further evaluate efficacy in this genetic eye condition. During the study, participants will follow scheduled visits, treatments, and laboratory tests for up to one year after receiving FT-002. Researchers will monitor safety and tolerance primarily, along with measuring efficacy outcomes. Participants will be closely monitored for any effects of the gene therapy, with assessments during this one-year period to understand the treatment's impact.
CONDITIONS
Brief Title
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male subjects willing and able to follow study procedures and sign informed consent
- Age 18 to 45 years at time of consent for Phase I dose escalation
- Age 8 to 45 years at time of consent for Phase II dose expansion
- Clinically diagnosed with X-linked retinitis pigmentosa, showing symptoms such as night blindness and vision loss
You will not qualify if you...
- Presence of other retinal degenerative diseases caused by other inherited gene variants
- Prior treatment with any gene therapy product
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 year
Participants receive a single intraocular injection of FT-002 gene therapy.
Multiple follow-up visits during the year after injection
Trial Site Locations
Total: 1 location
1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100142
Actively Recruiting
Research Team
X
Xinyan Li
M
Minghui Xue
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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