Identifying potential participants for Stargardt disease clinical trials
Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials
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 |
|---|---|
| Enrollment | 50 (estimated) |
| Ages | 5 Years and up |
| Sex | All |
| Sponsor | Ascidian Therapeutics, Inc Industry-sponsored |
| Locations | 8 sites (San Francisco, California and 7 other locations) |
| Trial ID | NCT06445322 on ClinicalTrials.gov |
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.
Who should consider this trial
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.
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.
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 performed: Other studies focusing on prescreening for genetic conditions have shown success in improving participant recruitment and trial efficiency, indicating a promising approach.
Eligibility criteria
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 this trial is running
San Francisco, California and 7 other locations
- University of San Francisco — San Francisco, California, United States (Recruiting)
- Vitreo Retinal Associates — Gainesville, Florida, United States (Recruiting)
- Wilmer Eye Institute at John Hopkins — Baltimore, Maryland, United States (Recruiting)
- Massachusetts Eye and Ear — 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)
- Retina Consultants of Texas — Houston, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Associate Director, Clinical Operations
- Email: researchtrials@ascidian-tx.com
- Phone: 207-573-0412
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.