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Trial record 4 of 17 for:    choroideremia

"Natural History" Study of Choroideremia

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2016 by 4D Molecular Therapeutics
Information provided by (Responsible Party):
4D Molecular Therapeutics Identifier:
First received: December 13, 2016
Last updated: May 18, 2017
Last verified: December 2016
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

Condition Intervention
Other: Observation

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Multicenter Prospective Observational "Natural History" Study in Patients With Choroideremia

Resource links provided by NLM:

Further study details as provided by 4D Molecular Therapeutics:

Primary Outcome Measures:
  • Rate of progression of disease [ Time Frame: 2 years ]
    A variety of psychophysical, anatomical and image-based endpoints

Estimated Enrollment: 50
Study Start Date: December 2016
Estimated Study Completion Date: December 2019
Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
No intervention
Other: Observation


Ages Eligible for Study:   14 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Individuals with a clinical diagnosis of choroideremia

Inclusion Criteria:

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

Exclusion Criteria:

  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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02994368

Contact: Jenny Holt (510) 505-2680

United States, California
Retina and Vitreous Associates of Los Angeles Recruiting
Los Angeles, California, United States
Contact: Janet Kurokouchi    310-289-2478 ext 1243   
Principal Investigator: David Liao, MD         
United States, Texas
Retina Foundation of the Southwest Recruiting
Dallas, Texas, United States, 75251
Contact: Kirsten Locke    214-363-3911   
Principal Investigator: David Birch, PhD         
United States, Utah
Moran Eye Center, University of Utah Recruiting
Salt Lake City, Utah, United States, 84132
Contact: Katie Rogers    801-581-2352   
Principal Investigator: Paul Bernstein, MD         
Sponsors and Collaborators
4D Molecular Therapeutics
Study Chair: Peter J Francis, MD PhD 4D Molecular Therapeutics
  More Information

Responsible Party: 4D Molecular Therapeutics Identifier: NCT02994368     History of Changes
Other Study ID Numbers: 4D-CHM-001-NH-0001
Study First Received: December 13, 2016
Last Updated: May 18, 2017
Individual Participant Data  
Plan to Share IPD: Yes

Keywords provided by 4D Molecular Therapeutics:
retinal degeneration
REP-1 gene

Additional relevant MeSH terms:
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked processed this record on May 24, 2017