4D-125 FOR THE TREATMENT OF XLRP

Study title

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

Status

Recruiting

About the study

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.

Who can participate?
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.

CHM NATURAL HISTORY STUDY

Study title

A Multicenter Prospective Observational “Natural History” Study in Patients With Choroideremia (CHM)

Status

Recruiting

About the study

The purpose of this study is to understand the rate of progression of all stages of CHM using a variety of assessments performed in the clinic including visual field measures, specialized photography of the eye and participant-reported visual problems.

Who can participate?
Ages eligible for study :

≥14 years (Primary Cohort)

≥18 years (Expansion Cohort)

Sexes eligible for study: Male
Accepts healthy volunteers: No

If you are interested in learning more about this study, visit clinicaltrials.gov for a list of active sites and contact the site’s study coordinator for more information.

4D-110 FOR THE TREATMENT OF CHM

Study title

Phase 1/2 Open-label, Dose-escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia (CHM)

Status

Active, not recruiting

About the study

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.

Who can participate?
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.

4D-310 FOR THE TREATMENT OF FABRY DISEASE

Study title

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males With Fabry Disease

Status

Recruiting

About the study

This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability and pharmacodynamics of 4D-310 following a single IV administration.

Who can participate?
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.