Actively Recruiting
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
Led by Frontera Therapeutics · Updated on 2024-07-09
32
Participants Needed
1
Research Sites
95 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase).
CONDITIONS
Official 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 including scheduled visits, treatment plan, and laboratory tests, and sign informed consent
- Age 18 to 45 years old at time of consent for Phase I dose escalation stage
- Age 8 to 45 years old at time of consent for Phase II dose expansion stage
- Clinically diagnosed with X-linked retinitis pigmentosa (XLRP) with symptoms such as night blindness, visual field loss, or vision loss
You will not qualify if you...
- Presence of other retinal degenerative diseases caused by known inherited retinal disease gene variants
- Prior receipt of any gene therapy product
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100142
Actively Recruiting
Research Team
X
Xinyan Li
CONTACT
M
Minghui Xue
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here