"Natural History" Study of Choroideremia

4D Molecular Therapeutics (4DMT)

Status

Terminated

Conditions

Choroideremia

Treatments

Other: Observation

Study type

Observational

Funder types

Industry

Identifiers

NCT02994368

4D-CHM-001-NH-0001

Details and patient eligibility

About

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

Enrollment

57 patients

Sex

Male

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria (Primary Cohort):

Healthy individuals (at least 14 years) with choroideremia (20/200 or better vision) willing to participate in an observational study and meeting the eligibility criteria.

Inclusion Criteria (Expansion Cohort):

Healthy individuals (at least 18 years) with choroideremia (20/200 or better vision in both eyes) willing to participate in an observational study and meeting the eligibility criteria.

Exclusion Criteria (Primary and Expansion Cohort):

Prior therapy with an AAV vector-based treatment
Pre-existing eye conditions that would: (1) preclude future planned treatment in a therapeutic intent clinical trial (i.e. intravitreal injection), (2) interfere with the interpretation of study endpoints, and/ or put patient at risk for surgical complications
Complicating systemic diseases that would preclude future enrollment in a therapeutic intent clinical trial
Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.