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"Natural History" Study of Choroideremia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02994368
Recruitment Status : Terminated
First Posted : December 15, 2016
Last Update Posted : January 6, 2022
Sponsor:
Information provided by (Responsible Party):
4D Molecular Therapeutics

Study Description
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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

Condition or disease Intervention/treatment
Choroideremia Other: Observation

Study Design
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Study Type : Observational
Actual Enrollment : 57 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Multicenter Prospective Observational "Natural History" Study in Patients With Choroideremia
Study Start Date : December 2016
Actual Primary Completion Date : October 2021
Actual Study Completion Date : October 2021

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Primary Cohort
No intervention
 Other: Observation
Expansion Cohort
No intervention
 Other: Observation


Outcome Measures
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Primary Outcome Measures :
  1. Rate of progression of disease [ Time Frame: 4 years ]
    A variety of psychophysical, anatomical and image-based endpoints


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Individuals with a clinical diagnosis of choroideremia
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02994368


Locations
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United States, Texas
Retina Foundation of the Southwest
Dallas, Texas, United States, 75251
Baylor College of Medicine, Ophthalmology-Cullen Eye Center
Houston, Texas, United States, 77030
United States, Utah
Moran Eye Center, University of Utah
Salt Lake City, Utah, United States, 84132
Canada, Alberta
University of Alberta
Edmonton, Alberta, Canada
Sponsors and Collaborators
4D Molecular Therapeutics
Investigators
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Study Director: Schonmei Lee, MD 4D Molecular Therapeutics
More Information
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Responsible Party: 4D Molecular Therapeutics
ClinicalTrials.gov Identifier: NCT02994368    
Other Study ID Numbers: 4D-CHM-001-NH-0001
First Posted: December 15, 2016    Key Record Dates
Last Update Posted: January 6, 2022
Last Verified: January 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by 4D Molecular Therapeutics:
retinal degeneration
eye
REP-1 gene
Additional relevant MeSH terms:
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Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
 Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked