Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

Launched by FRONTERA THERAPEUTICS · Jul 1, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called FT-002 for treating a specific type of vision loss known as X-linked retinitis pigmentosa (XLRP), which is caused by a mutation in the RPGR gene. The goal is to see if a one-time injection of this therapy into the eye is safe, well-tolerated, and effective in improving vision for male participants aged 8 to 45 years old. The trial is currently recruiting participants, and it will take place in two stages: the first stage will test different doses to find the best one, and the second stage will focus on a larger group of participants.

To be eligible for this trial, participants must be male, aged 8 to 45, and have a confirmed diagnosis of XLRP, which often leads to symptoms like night blindness and loss of vision. They should also be willing to follow the study procedures and attend regular visits. However, people with other eye diseases or those who have previously received gene therapy for eye conditions cannot participate. If you or someone you know is interested, participants will receive detailed information about what to expect throughout the study, including visits for tests and monitoring after the injection.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Subjects that are willing and able to follow study procedures including scheduled visits, treatment plan, and laboratory tests, and sign a written informed consent form;
• • Age: Phase I dose escalation stage, 18-45 years old male (including boundary value) at the time of signing the ICF; Phase II dose extension stage, males 8-45 years old (including boundary values) at the time of signing the ICF;
• • Clinically diagnosed XLRP, the main symptoms include but are not limited to night blindness, visual field loss, vision loss, etc.;
• Exclusion Criteria:
• • 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.