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

Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials

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.

Study Overview

• Status: Recruiting
• Conditions: Stargardt Disease; Stargardt Disease 1; Juvenile Macular Degeneration; Cone Rod Dystrophy
• Intervention / Treatment: Diagnostic test: Prescreening Assessments

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 Type: Observational
• Enrollment (Estimated): 50

Contacts and Locations

• Study Contact: Name: Associate Director, Clinical Operations; Phone Number: 207-573-0412; Email: researchtrials@ascidian-tx.com

Study Locations

• United States
  • California
    • San Francisco, California, United States, 94158
      • Recruiting
      • University of San Francisco
  • Florida
    • Gainesville, Florida, United States, 32607
      • Recruiting
      • Vitreo Retinal Associates
  • Maryland
    • Baltimore, Maryland, United States, 21218
      • Recruiting
      • Wilmer Eye Institute at John Hopkins
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Recruiting
      • Massachusetts Eye and Ear
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Recruiting
      • University of Michigan Kellogg Eye Center
  • Ohio
    • Cincinnati, Ohio, United States, 45245
      • Recruiting
      • Cincinnati Eye Institute
  • Texas
    • Dallas, Texas, United States, 75382
      • Recruiting
      • Retina Foundation of Texas
    • Houston, Texas, United States, 77401
      • Recruiting
      • Retina Consultants of Texas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Males and females with a diagnosis of ABCA4-related retinopathy will be invited to give informed consent prior to prescreening procedures. After appropriate informed consent/assent has been obtained, potential participants will then undergo the assessments to determine eligibility for ACDN-01 clinical trials.

Description

Key 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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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.	Diagnostic test: Prescreening Assessments; Various genetic and visual assessments.

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Ascidian Therapeutics, Inc
• Investigators: Study Director: Alia Rashid, Ascidian Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): June 20, 2024
• Primary Completion (Estimated): August 31, 2027
• Study Completion (Estimated): August 31, 2030

Study Registration Dates

• First Submitted: May 31, 2024
• First Submitted That Met QC Criteria: May 31, 2024
• First Posted (Actual): June 6, 2024

Study Record Updates

• Last Update Posted (Actual): March 11, 2026
• Last Update Submitted That Met QC Criteria: March 9, 2026
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: Gene Therapy; RNA; IRD; Stargardt Disease; ABCA4; Inherited retinal disease; Stargardt macular dystrophy; Gene editing; Inherited retinal dystrophy; ABCA4-related retinopathy; Cone rod dystrophy; Exon editing; Inherited retinal degeneration
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases; Eye Diseases, Hereditary; Retinal Diseases; Retinal Dystrophies; Retinal Degeneration; Macular Degeneration; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cone-Rod Dystrophies; Stargardt Disease
• Other Study ID Numbers: ACDN-01-000

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No