Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials
Overview
- Phase
- Not Applicable
- Intervention
- Prescreening Assessments
- Conditions
- Stargardt Disease
- Sponsor
- Ascidian Therapeutics, Inc
- Enrollment
- 50
- Locations
- 8
- Primary Endpoint
- Confirm mutations in the ABCA4 gene
- Status
- Recruiting
- Last Updated
- last month
Overview
Brief Summary
This is an observational prescreening study. Individuals who are eligible for prescreening will undergo testing procedures that may be used to determine eligibility in ACDN-01 clinical trials.
Detailed Description
The prescreening process will be used to help determine the initial eligibility and interest of potential participants in ACDN-01 clinical trials by conducting assessments of key eligibility criteria before the clinical trial screening procedures are performed.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Presence of mutations in the ABCA4 gene
- •ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
Exclusion Criteria
- •The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy
- •Retinal disease other than ABCA4-related retinopathy
- •Presence of a medical condition (systemic or ophthalmic), psychiatric condition, including substance abuse disorder, or physical examination or laboratory finding that may in the opinion of the principal investigator and sponsor preclude adherence to the scheduled study visits, safe participation in the study, or affect the results of the study.
Arms & Interventions
Prescreening Group
The prescreening study consists of genetic and visual assessments and will require at least 1 onsite visit. All clinical assessments performed are for the purpose of determining research eligibility for ACDN-01 clinical trials.
Intervention: Prescreening Assessments
Outcomes
Primary Outcomes
Confirm mutations in the ABCA4 gene
Time Frame: 12 months
Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.
Measure BCVA and LLVA
Time Frame: 12 months
Measure best corrected visual acuity and low luminance visual acuity
Measure the area of retinal atrophy
Time Frame: 12 months
Using FAF imaging
Measure baseline retinal structure
Time Frame: 12 months
Using OCT (SD-OCT)
Historical FAF or OCT images
Time Frame: 4 years
Confirm historical timepoint images
Confirm the absence of pathogenic mutations in genes known to cause retinal disease other than ABCA4-related retinopathy
Time Frame: 12 months
Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.
Historical BCVA/LLVA measurements
Time Frame: 4 years
Collect past measurements