Actively Recruiting

Age: 5Years +
All Genders
ID06445322

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

Led by Ascidian Therapeutics, Inc · Updated on 2026-03-11

50

Participants Needed

8

Research Sites

156 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This observational prescreening study focuses on individuals with Stargardt disease and related ABCA4 gene mutations. It aims to identify potential participants for future ACDN-01 clinical trials by assessing key eligibility criteria before formal trial screening. The study is sponsored by Ascidian Therapeutics, Inc and targets people with ABCA4-related retinopathy, including Stargardt disease type 1 and cone-rod dystrophy. Participants will undergo genetic and visual assessments during at least one onsite visit. These tests include confirming mutations in the ABCA4 gene, measuring visual acuity (BCVA and LLVA), retinal atrophy area, and retinal structure. Historical imaging and vision test data may also be reviewed to support eligibility evaluation. During the study, researchers will collect data on the participant's genetic status and eye health to determine suitability for ACDN-01 clinical trials. Assessments involve genetic testing, vision measurements, and retinal imaging. The primary outcomes include confirmation of gene mutations and detailed eye examinations over a 12-month period, with some historical data collected for up to 4 years. Participants' safety and adherence to study visits are monitored throughout the process.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 5Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Presence of mutations in the ABCA4 gene
  • ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
Not Eligible

You will not qualify if you...

  • 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
  • Medical condition (systemic or ophthalmic), psychiatric condition including substance abuse disorder, or examination or lab findings that may affect safe participation or study adherence

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Diagnostic Evaluation

Duration - Up to 12 months

Participants undergo genetic and visual assessments to determine eligibility for further ACDN-01 clinical trials.

At least 1 onsite visit

Long-term Monitoring

Duration - Up to 4 years

Participants' historical imaging and visual acuity data are reviewed to support eligibility assessment.

No visits required; data collected from medical history

Trial Site Locations

Total: 8 locations

1

University of San Francisco

San Francisco, California, United States, 94158

Actively Recruiting

2

Vitreo Retinal Associates

Gainesville, Florida, United States, 32607

Actively Recruiting

3

Wilmer Eye Institute at John Hopkins

Baltimore, Maryland, United States, 21218

Actively Recruiting

4

Massachusetts Eye and Ear

Boston, Massachusetts, United States, 02114

Actively Recruiting

5

University of Michigan Kellogg Eye Center

Ann Arbor, Michigan, United States, 48105

Actively Recruiting

6

Cincinnati Eye Institute

Cincinnati, Ohio, United States, 45245

Actively Recruiting

7

Retina Foundation of Texas

Dallas, Texas, United States, 75382

Actively Recruiting

8

Retina Consultants of Texas

Houston, Texas, United States, 77401

Actively Recruiting

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Research Team

A

Associate Director, Clinical Operations

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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