Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
Stargardt Disease · Cone Rod Dystrophy · Juvenile Macular Degeneration · Stargardt Disease 1
What this study involves
The registry does not currently label this study as recruiting. Ask about its status before planning a screening visit.
- Study type
- Interventional
- Phase
- Phase 1 / Phase 2
- Age range
- 12 years - Not specified
- Sex eligible
- All sexes
- Healthy volunteers
- Not accepted
- Participants
- 15 Estimated
These are starting points, not an eligibility decision. The research team must review the full requirements.
About the research
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.
Read the full description
This is an open-label, single ascending dose study of ACDN-01 in adult participants with ABCA4-related retinopathy. The study is designed to evaluate the safety, tolerability, and evidence of biological effect of SAD levels (low, medium, and high) of ACDN-01 when delivered subretinally.
Participants will be followed on study for 2 years for the primary safety and preliminary efficacy endpoints, after which they will continue in the study in a 3-year long-term follow-up period, for a total study duration of 5 years.
Who may be able to participate?
The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.
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.
Treatment, groups and participation
The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.
- Assignment
- Not randomized
- Masking
- No masking
Low dose of ACDN-01
One time low dose of ACDN-01.
Drug: ACDN-01
Mid-dose of ACDN-01
One time mid-dose of ACDN-01.
Drug: ACDN-01
High dose of ACDN-01
One time high dose of ACDN-01.
Drug: ACDN-01
ACDN-01 DRUG
ACDN-01 is an AAV-based vector carrying a DNA construct encoding for an ABCA4 RNA exon editor. One time administration is via subretinal injection.
What researchers are measuring
Outcome measures describe what is being studied. They are not proof that a treatment works.
Primary measures
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
Time frame: 12 months
To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy.
Secondary measures
Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.
Time frame: 12 months
To determine maximum tolerated dose (MTD) for subsequent clinical evaluation.
To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).
Time frame: 24 months
To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in the area of macular atrophy in mm2 as measured on FAF over 24 months.
To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).
Time frame: 24 months
To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in change in thickness in retinal layers as measured on OCT, over 24 months.
Other measures
To assess for evidence of preliminary efficacy of ACDN-01 based on best corrected visual acuity.
Time frame: 24 months
Best Corrected Visual Acuity is measured by number of ETDRS letters read in each eye compared to baseline.
To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures.
Time frame: 24 months
Patient-reported outcome measures will be measured by overall and subscale scores on the Michigan Retinal -Degeneration Questionnaire (MRDQ).
To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
Time frame: 24 months
Contrast sensitivity is measured using qCSF tests and will measure number of letters read and change in AUC (area under curve).
To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
Time frame: 24 months
Contrast sensitivity is measured using Pelli-Robson tests and will measure number of letters read and change in AUC (area under curve).
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
Time frame: 24 months
Retinal sensitivity will be measured by change in the location of detectable points.
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
Time frame: 24 months
Retinal sensitivity will be measured by change in the intensity (dB) of detectable points.
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
Time frame: 24 months
Retinal sensitivity will be measured by change in the number of detectable points.
Review the complete result tables and limitations in the official record.
Open official results ↗Research locations (11)
The overall study status and a local site’s status can differ. Confirm that location’s availability before traveling.
11 locations
University of San FranciscoSan Francisco, California, United StatesStatus unknown
Vitreo Retinal AssociatesGainesville, Florida, United StatesStatus unknown
Bascom Palmer Eye InstituteMiami, Florida, United StatesStatus unknown
Wilmer Eye Institute at John HopkinsBaltimore, Maryland, United StatesStatus unknown
Massachusetts Eye and EarBoston, Massachusetts, United StatesStatus unknown
University of Michigan Kellogg Eye CenterAnn Arbor, Michigan, United StatesStatus unknown
Cincinnati Eye InstituteCincinnati, Ohio, United StatesStatus unknown
Casey Eye Institute OHSUPortland, Oregon, United StatesStatus unknown
Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania, United StatesStatus unknown
Retina Foundation of the SouthwestDallas, Texas, United StatesStatus unknown
Retina Consultants of TexasHouston, Texas, United StatesStatus unknown
Study team and timeline
- Lead sponsor
- Ascidian Therapeutics, Inc
- Study start
- Jun 11, 2024 Actual
- Primary completion
- Aug 1, 2030 Estimated
- Study completion
- Dec 1, 2030 Estimated
- First posted
- Jun 21, 2024 Actual
- Last updated
- Nov 10, 2026 Actual
Official study contacts
No central contact is listed. Check the locations and the official record.
Official study title
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
Ask Wellness Clinical Research
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Source: ClinicalTrials.gov API v2. Information is submitted by sponsors and investigators. NLM does not approve the safety or science of all listed studies. Registry disclaimer.
Retrieved: Nov 12, 2026 · Last registry update: Nov 10, 2026