Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Prescreening Assessments.
- Who it may be relevant to
- Registry conditions: Stargardt Disease, Stargardt Disease 1, Cone Rod Dystrophy, Juvenile Macular Degeneration. Basic parameters: from 5 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials
Overview
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.
Interventions
- Diagnostic test Prescreening Assessments
Various genetic and visual assessments.
Primary outcome measures
- Confirm mutations in the ABCA4 gene [Time frame: 12 months]
- Confirm the absence of pathogenic mutations in genes known to cause retinal disease other than ABCA4-related retinopathy [Time frame: 12 months]
- Measure BCVA and LLVA [Time frame: 12 months]
- Measure the area of retinal atrophy [Time frame: 12 months]
- Measure baseline retinal structure [Time frame: 12 months]
- Historical FAF or OCT images [Time frame: 4 years]
- Historical BCVA/LLVA measurements [Time frame: 4 years]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 8 centers
- University of San Francisco — San Francisco
- Vitreo Retinal Associates — Gainesville
- Wilmer Eye Institute at John Hopkins — Baltimore
- Massachusetts Eye and Ear — Boston
- University of Michigan Kellogg Eye Center — Ann Arbor
- Cincinnati Eye Institute — Cincinnati
- Retina Foundation of Texas — Dallas
- Retina Consultants of Texas — Houston
Identifiers
NCT: NCT06445322 · ACDN-01-000