Choroideremia Health Outcomes
Data Collection
Collected from today forward - ProspectiveEye Diseases+5
+ Congenital, Hereditary, and Neonatal Diseases and Abnormalities
+ Uveal Diseases
Cohort
Tracking disease incidence in order to identify risk factors and understand disease progression over time.Summary
Study start date: December 15, 2020
Actual date on which the first participant was enrolled.The primary objectives of this study are to describe disease progression and severity by age in participants with CHM, to assess health-related quality-of-life, resource utilization and work productivity, and to assess quality-of-life, work productivity, and impact on daily activities in caregivers of participants with CHM at different stages of disease progression.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.46 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Cohort
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Male
Biological sex of participants that are eligible to enroll.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
Key Inclusion Criteria: Physician Participants 1. At least 60% of time spent in direct participant care. 2. Board-certified or eligible with a Specialty in Ophthalmology, such as Retinal Specialist, IRD Specialist, Retinal Surgeon. Patient Participants 1. CHM diagnosis confirmed via genetic testing. 2. Include any minimal disease severity requirement. 3. Participant has at least one record of visual acuity measurement in the past 24 months, OR has be assigned by the treating clinician as having severe VA impairment of blind/legally blind by using one of the following methods: finger counting, hand movement, light perception / no light perception. Key Exclusion Criteria: Physician Participants a. Participating physicians must not be affiliated with an approved management organization. Patient Participants a. Participants who received gene therapy or any other investigational treatment and participants who do not agree to informed consent. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.Study Objectives
Primary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 8 locations
Research Site
San Francisco, United StatesResearch Site
Coral Gables, United StatesResearch Site
Gainesville, United States