| Ages eligible for study : | 50 Years and older (Adult, Older Adult) |
| Sexes eligible for study: | All |
| Accepts healthy volunteers: | No |
If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for a list of active sites and contact the site’s study coordinator for more information.
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males With X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR Gene
Active, not recruiting
This Phase 1/2 study will gather data in an observational phase Natural History Cohort to further characterize and evaluate natural disease progression in male patients with genetically-confirmed XLRP caused by mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR). The study will also evaluate the safety and tolerability, as assessed by frequency and severity of ocular and systemic adverse events, as well as preliminary clinical efficacy of a single intravitreal (IVT) injection of 4D-125 at two dose levels in this patient population.
| Ages eligible for study : | ≥6 years (Natural History Cohort)
≥18 years (Interventional Cohort) |
| Sexes eligible for study: | Male |
| Accepts healthy volunteers: | No |
If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for a list of active sites and contact the site’s study coordinator for more information.
Phase 1/2 Open-label, Dose-escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia (CHM)
Active, not recruiting
This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single intravitreal (IVT) injection of 4D-110 at two dose levels in male patients with genetically-confirmed CHM.
| Ages eligible for study : | ≥18 years |
| Sexes eligible for study: | Male |
| Accepts healthy volunteers: | No |
If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for a list of active sites and contact the site’s study coordinator for more information.
| Ages eligible for study : | 50 Years and older (Adult, Older Adult) |
| Sexes eligible for study: | All |
| Accepts healthy volunteers: | No |
If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for a list of active sites and contact the site’s study coordinator for more information.
| Ages eligible for study : | 18 years |
| Maximum Age: | |
| Sexes eligible for study: | All |
| Accepts healthy volunteers: | No |
If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for a list of active sites and contact the site’s study coordinator for more information.