"Natural History" Study of Choroideremia

NCT02994368 | Terminated

• 1 other identifier: 4D-CHM-001-NH-0001
• Study Type: observational
• Target: 57
• Locations: 2 countries
• Sites: 4

Brief 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

Conditions

Choroideremia

Keywords

retinal degeneration; eye; REP-1 gene

Outcome Measures

Primary Outcomes (1)

• Rate of progression of disease

  A variety of psychophysical, anatomical and image-based endpoints

  4 years

Study Arms (2)

Primary Cohort

No intervention

Other: Observation

Expansion Cohort

No intervention

Other: Observation

Interventions

Observation OTHER

Expansion Cohort; Primary Cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Individuals with a clinical diagnosis of choroideremia

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• 4D Molecular Therapeutics: lead

Study Sites (4)

Retina Foundation of the Southwest

Dallas, Texas, 75251, United States

Location

Baylor College of Medicine, Ophthalmology-Cullen Eye Center

Houston, Texas, 77030, United States

Location

Moran Eye Center, University of Utah

Salt Lake City, Utah, 84132, United States

Location

University of Alberta

Edmonton, Alberta, Canada

Location

MeSH Terms

Conditions

Choroideremia; Retinal Degeneration

Interventions

Observation

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Choroid Diseases; Uveal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked; Retinal Diseases

Intervention Hierarchy (Ancestors)

Methods; Investigative Techniques

Study Officials

• Schonmei Lee, MD

  4D Molecular Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 13, 2016
• First Posted: December 15, 2016
• Study Start: December 1, 2016
• Primary Completion: October 1, 2021
• Study Completion: October 1, 2021
• Last Updated: January 6, 2022

Record last verified: 2022-01

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (3)