Identifying potential participants for Stargardt disease clinical trials
Official title: Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials
Run by Ascidian Therapeutics, Inc · NCT06445322 · Data updated 2026-11-12
This study is trying to find people with Stargardt disease who might qualify for upcoming clinical trials by checking if they meet certain criteria, like having specific gene mutations.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 5 Years and up |
| Sex | All |
| Enrollment | 50 (estimated) |
| Sponsor | Ascidian Therapeutics, Inc Industry-sponsored |
| Locations | 9 sites (San Francisco, California and 8 other locations) |
| Status | Recruiting |
| Started | June 20, 2024 |
| Main results expected | August 31, 2027 |
| Study ends | August 31, 2030 |
| Registry record updated | October 9, 2026 |
| Trial ID | NCT06445322 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are individuals with mutations in the ABCA4 gene and a phenotype of ABCA4-related retinopathy, such as Stargardt disease type 1 or cone-rod dystrophy.
✕Probably not a fit
Patients with retinal diseases caused by mutations in genes other than ABCA4 or those with other significant medical conditions may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational prescreening study aims to identify individuals who may qualify for the ACDN-01 clinical trials focused on Stargardt disease and related conditions. Eligible participants will undergo specific testing procedures to assess their suitability based on key eligibility criteria, including the presence of mutations in the ABCA4 gene. The prescreening process is designed to streamline the recruitment of participants for future clinical trials by determining their initial eligibility and interest. This approach allows for a more efficient selection of candidates for the subsequent phases of the clinical trials.
Why it matters
Potential benefit: If successful, this study could enhance the recruitment process for clinical trials targeting Stargardt disease, potentially leading to new treatment options.
How similar studies have gone: Other studies focusing on prescreening for genetic conditions have shown success in improving participant recruitment and trial efficiency, indicating a promising approach.
Who can join: the full rules
Show full inclusion / exclusion criteria
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.
Where it's running
-
Vitreo Retinal Associates
Gainesville, Florida, United States Recruiting -
Massachusetts Eye and Ear
Boston, Massachusetts, United States Recruiting -
Boston Children's Hospital
Boston, Massachusetts, United States Recruiting -
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, United States Recruiting -
Cincinnati Eye Institute
Cincinnati, Ohio, United States Recruiting -
Retina Foundation of Texas
Dallas, Texas, United States Recruiting
Show 3 more locations
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Retina Consultants of Texas
Houston, Texas, United States Recruiting -
University of San Francisco
San Francisco, California, United States Terminated -
Wilmer Eye Institute at John Hopkins
Baltimore, Maryland, United States Terminated
Study contacts
- Study coordinator: Senior Clinical Trial Manager
- Email: researchtrials@ascidian-tx.com
- Phone: 207-573-0412
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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City-RBP4 in healthy people and Stargardt disease type 1 patients
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ASP2020 eye injection safety in Stargardt-type macular dystrophy
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A first-in-human study will test the safety and tolerability of a single subretinal injection of C.001 in adults with retinal diseases involving retinal pigment epithelium degeneration.
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This study will see if a 30-minute session of transcorneal electrical stimulation with the OkuStim device produces short-term vision improvements in adults with retinitis pigmentosa or similar retinal degenerations who already use the device.
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Functional visual field and navigation testing for moderate-to-profound inherited retinal disease
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.