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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 : Active, not recruiting
First Posted : December 15, 2016
Last Update Posted : December 7, 2020
Sponsor:
Collaborator:
Roche Pharma AG
Information provided by (Responsible Party):
4D Molecular Therapeutics

Tracking Information
First Submitted Date December 13, 2016
First Posted Date December 15, 2016
Last Update Posted Date December 7, 2020
Study Start Date December 2016
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 14, 2020)
Rate of progression of disease [ Time Frame: 4 years ]
A variety of psychophysical, anatomical and image-based endpoints
Original Primary Outcome Measures
 (submitted: December 14, 2016)
Rate of progression of disease [ Time Frame: 2 years ]
A variety of psychophysical, anatomical and image-based endpoints
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title "Natural History" Study of Choroideremia
Official Title A Multicenter Prospective Observational "Natural History" Study in Patients With Choroideremia
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
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Individuals with a clinical diagnosis of choroideremia
Condition Choroideremia
Intervention Other: Observation
Study Groups/Cohorts Observation
No intervention
Intervention: Other: Observation
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Estimated Enrollment
 (submitted: December 14, 2016)
50
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 2022
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.
Sex/Gender
Sexes Eligible for Study: Male
Ages 14 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02994368
Other Study ID Numbers 4D-CHM-001-NH-0001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party 4D Molecular Therapeutics
Study Sponsor 4D Molecular Therapeutics
Collaborators Roche Pharma AG
Investigators
Study Director: Robert Kim, MD 4D Molecular Therapeutics
PRS Account 4D Molecular Therapeutics
Verification Date December 2020