Evaluating ACDN-01 for treating Stargardt Retinopathy
ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy
This study is testing a new treatment called ACDN-01 to see if it can help adults with Stargardt disease and related eye conditions feel better and improve their vision.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | Ascidian Therapeutics, Inc Industry-sponsored |
| Locations | 10 sites (San Francisco, California and 9 other locations) |
| Trial ID | NCT06467344 on ClinicalTrials.gov |
What this trial studies
This clinical trial is an open-label, single ascending dose study assessing the safety, tolerability, and preliminary efficacy of ACDN-01 in adults with ABCA4-related retinopathies, specifically Stargardt disease and cone-rod dystrophy. Participants will receive a single subretinal injection of ACDN-01 and will be monitored for two years for primary safety and efficacy endpoints, followed by an additional three years of long-term follow-up. The study aims to determine the biological effects of varying doses of ACDN-01 delivered subretinally.
Who should consider this trial
Good fit: Ideal candidates for this study are adults with confirmed mutations in the ABCA4 gene and a diagnosis of 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 significant comorbid conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from ABCA4-related retinopathies, potentially improving vision and quality of life.
How similar studies have performed: While this approach is novel in the context of ABCA4-related retinopathies, similar gene therapies have shown promise in other retinal diseases.
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) * Area of atrophy located in the macula of the study eye * BCVA of 20/50 (0.4 logMAR) or worse 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 9 other locations
- University of San Francisco — San Francisco, California, United States (Recruiting)
- Vitreo Retinal Associates — Gainesville, Florida, United States (Recruiting)
- Bascom Palmer Eye Institute — Miami, 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)
- Casey Eye Institute OHSU — Portland, Oregon, United States (Recruiting)
- Retina Foundation of the Southwest — 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.