“Natural History” Study of Choroideremia

Overview

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

Full Title of Study: “A Multicenter Prospective Observational “Natural History” Study in Patients With Choroideremia”

Study Type

  • Study Type: Observational
  • Study Design
    • Time Perspective: Prospective
  • Study Primary Completion Date: June 2022

Interventions

  • Other: Observation

Arms, Groups and Cohorts

  • Primary Cohort
    • No intervention
  • Expansion Cohort
    • No intervention

Clinical Trial Outcome Measures

Primary Measures

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

Participating in This Clinical Trial

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.

Gender Eligibility: Male

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • 4D Molecular Therapeutics
  • Collaborator
    • Roche Pharma AG
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Robert Kim, MD, Study Director, 4D Molecular Therapeutics
  • Overall Contact(s)
    • Paula Lea, 510-505-2680, clinicaltrials@4dmt.com

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