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Study of ACDN-01 for ABCA4-related Stargardt Retinopathy

This study is testing a new treatment called ACDN-01 for people with Stargardt disease or cone-rod dystrophy caused by changes in the ABCA4 gene. ACDN-01 is given as a single injection into the eye. The main goal of this study is to see if ACDN-01 is safe and how well people tolerate it over 12 months. You might be able to join if you are 12 years or older, have specific changes in your ABCA4 gene, and your vision is 20/50 or worse in the study eye. This is the first time ACDN-01 is being tested in people. The study is currently recruiting 15 participants, but its overall status is unclear.

Study design
This is an open-label study, meaning everyone knows what treatment is being given. It will test different dose levels of ACDN-01 in a small group of 15 participants.
What's involved
Participants will be followed for 2 years for safety and initial effectiveness, then for an additional 3 years for long-term follow-up, totaling 5 years.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 5 years after treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06467344

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

Recruiting
PHASE1Ages 12+InterventionalTreatment
Ascidian Therapeutics, Inc
~15 participants
Updated 2025-12-02 on ClinicalTrials.gov
What's tested:ACDN-01

At a glance

Recruiting sites
10 of 10 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
Measured over 12 months

Conditions

Stargardt Disease
Cone Rod Dystrophy
Juvenile Macular Degeneration
Stargardt Disease 1

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Where it's being run

10 sites across 8 states
Florida2
Texas2
California1
Maryland1
Massachusetts1
Michigan1
Ohio1
Oregon1

Study leadership

  • Alia Rashid · STUDY_DIRECTOR · Ascidian Therapeutics

Who to contact

Associate Director, Clinical Operations
[email protected]
207-573-0412
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

Exclusion

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.

What this trial measures

  • Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.12 months

    To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy.