"Natural History" Study of Choroideremia

Choroideremia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02994368
• Other study ID #: 4D-CHM-001-NH-0001
• Status: Terminated
• Start date: December 2016
• Est. completion date: October 2021

Study information

• Verified date: January 2022
• Source: 4D Molecular Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 57
• Est. completion date: October 2021
• Est. primary completion date: October 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Choroideremia

Intervention

Other:
• Observation

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
4D Molecular Therapeutics

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rate of progression of disease	A variety of psychophysical, anatomical and image-based endpoints	4 years