NCT06467344RecruitingPhase 1/2
Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
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
Source: ClinicalTrials.gov · Last updated 7 Oct 2026
Brief summary (registry, in English)
This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 15 (estimated)
- First submitted
- 31 May 2024
- Start date
- 11 Jun 2024
- Primary completion date
- 1 Aug 2030
- Lead sponsor
- Ascidian Therapeutics, Inc
- Diseases
- Macular degeneration, Retinitis pigmentosa
- NCT number
- NCT06467344
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 15
- From 11 Jun 2024 to the primary completion date, 1 Aug 2030
- 11 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study design.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Retinal disease other than ABCA4-related retinopathy
Lab values
- 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.
Other
- The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy
Who the study is looking for
- 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
Age in the registry: 12 years to - · Sex: All · Does not accept healthy volunteers
Sites (11)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Michigan Kellogg Eye Center Coordinator of this site? Confirm its status | Ann Arbor | United States | Recruiting |
| Wilmer Eye Institute at John Hopkins Coordinator of this site? Confirm its status | Baltimore | United States | Terminated |
| Massachusetts Eye and Ear Coordinator of this site? Confirm its status | Boston | United States | Recruiting |
| Cincinnati Eye Institute Coordinator of this site? Confirm its status | Cincinnati | United States | Recruiting |
| Retina Foundation of the Southwest Coordinator of this site? Confirm its status | Dallas | United States | Recruiting |
| Vitreo Retinal Associates Coordinator of this site? Confirm its status | Gainesville | United States | Recruiting |
| Retina Consultants of Texas Coordinator of this site? Confirm its status | Houston | United States | Recruiting |
| Bascom Palmer Eye Institute Coordinator of this site? Confirm its status | Miami | United States | Recruiting |
| Children's Hospital of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
| Casey Eye Institute - OHSU Coordinator of this site? Confirm its status | Portland | United States | Recruiting |
| University of San Francisco Coordinator of this site? Confirm its status | San Francisco | United States | Terminated |
This page is information, not medical advice. Discuss any trial with your doctor.