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

Launched by FRONTERA THERAPEUTICS · May 23, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new type of treatment called gene therapy for a condition known as X-linked retinitis pigmentosa (XLRP), which is a genetic eye disease that can lead to vision loss. The goal of the trial is to see if introducing a healthy copy of a specific gene can help improve or preserve vision in males who have a mutation in the RPGR gene, which is linked to this condition.

To participate in the trial, men aged 8 to 45 years old who have been diagnosed with XLRP and specifically have RPGR gene mutations may be eligible. It's important to note that participants should not have other eye diseases that cause similar problems. Those who join the study will follow specific procedures and may receive the gene therapy, with the hope of finding a new way to treat this challenging condition. If you or someone you know fits these criteria and is interested, this trial could be an opportunity to contribute to important research while exploring potential new treatment options.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • 1.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 ;
• Exclusion Criteria:
• • 1.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.