"Natural History" Study of Choroideremia

January 4, 2022 updated by: 4D Molecular Therapeutics

A Multicenter Prospective Observational "Natural History" Study in Patients With Choroideremia

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

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

57

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada
        • University of Alberta
  • United States
    • Texas
      • Dallas, Texas, United States, 75251
        • Retina Foundation of the Southwest
      • Houston, Texas, United States, 77030
        • Baylor College of Medicine, Ophthalmology-Cullen Eye Center
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • Moran Eye Center, University of Utah

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Non-Probability Sample

Study Population

Individuals with a clinical diagnosis of choroideremia

Description

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):

  1. Prior therapy with an AAV vector-based treatment
  2. 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
  3. Complicating systemic diseases that would preclude future enrollment in a therapeutic intent clinical trial
  4. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Primary Cohort
No intervention
Other: Observation
Expansion Cohort
No intervention
Other: Observation

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Rate of progression of disease
Time Frame: 4 years
A variety of psychophysical, anatomical and image-based endpoints
4 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

4D Molecular Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2016

Primary Completion (ACTUAL)

October 1, 2021

Study Completion (ACTUAL)

October 1, 2021

Study Registration Dates

First Submitted

December 13, 2016

First Submitted That Met QC Criteria

December 14, 2016

First Posted (ESTIMATE)

December 15, 2016

Study Record Updates

Last Update Posted (ACTUAL)

January 6, 2022

Last Update Submitted That Met QC Criteria

January 4, 2022

Last Verified

January 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 4D-CHM-001-NH-0001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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