Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials

Recruiting

• Conditions: Cone Rod Dystrophy; Juvenile Macular Degeneration; Stargardt Disease; Stargardt Disease 1
• Interventions: Diagnostic Test: Prescreening Assessments

• Registration Number: NCT06445322
• Lead Sponsor: Ascidian Therapeutics, Inc

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.

Study Timeline

• Study First Submitted: 2024-05-31
• Study First Submitted QC: 2024-05-31
• Study First Posted: 2024-06-06
• Study Start: 2024-06-20
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-12
• Last Update Posted: 2025-05-14
• Primary Completion: 2027-08-31
• Study Completion: 2030-08-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

• Presence of mutations in the ABCA4 gene
• ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)

Key

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Prescreening Group	Prescreening Assessments	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.

Primary Outcome Measures

Name	Time	Method
Confirm mutations in the ABCA4 gene	12 months	Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.
Measure the area of retinal atrophy	12 months	Using FAF imaging
Measure baseline retinal structure	12 months	Using OCT (SD-OCT)
Historical FAF or OCT images	4 years	Confirm historical timepoint images
Confirm the absence of pathogenic mutations in genes known to cause retinal disease other than ABCA4-related retinopathy	12 months	Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.
Measure BCVA and LLVA	12 months	Measure best corrected visual acuity and low luminance visual acuity
Historical BCVA/LLVA measurements	4 years	Collect past measurements

Trial Locations

Locations (8)

University of San Francisco; 🇺🇸 San Francisco, California, United States

Vitreo Retinal Associates; 🇺🇸 Gainesville, Florida, United States

Massachusetts Eye and Ear; 🇺🇸 Boston, Massachusetts, United States

Wilmer Eye Institute at John Hopkins; 🇺🇸 Baltimore, Maryland, United States

University of Michigan Kellogg Eye Center; 🇺🇸 Ann Arbor, Michigan, United States

Cincinnati Eye Institute; 🇺🇸 Cincinnati, Ohio, United States

Retina Foundation of Texas; 🇺🇸 Dallas, Texas, United States

Retina Consultants of Texas; 🇺🇸 Houston, Texas, United States