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

November 25, 2025 updated by: Ascidian Therapeutics, Inc

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 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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Estimated)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • San Francisco, California, United States, 94158
        • Recruiting
        • University of San Francisco
    • Florida
      • Gainesville, Florida, United States, 32607
        • Recruiting
        • Vitreo Retinal Associates
      • Miami, Florida, United States, 33136
        • Recruiting
        • Bascom Palmer Eye Institute
    • Maryland
      • Baltimore, Maryland, United States, 21218
        • Recruiting
        • Wilmer Eye Institute at John Hopkins
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts Eye and Ear
    • Michigan
      • Ann Arbor, Michigan, United States, 48105
        • Recruiting
        • University of Michigan Kellogg Eye Center
    • Ohio
      • Cincinnati, Ohio, United States, 45245
        • Recruiting
        • Cincinnati Eye Institute
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Casey Eye Institute OHSU
    • Texas
      • Dallas, Texas, United States, 75382
        • Recruiting
        • Retina Foundation of the Southwest
      • Houston, Texas, United States, 77401
        • Recruiting
        • Retina Consultants of Texas

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Low dose of ACDN-01
One time low dose of ACDN-01.
Drug: ACDN-01
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.
Experimental: Mid-dose of ACDN-01
One time mid-dose of ACDN-01.
Drug: ACDN-01
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.
Experimental: High dose of ACDN-01
One time high dose of ACDN-01.
Drug: ACDN-01
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 is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
12 months
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.
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.
24 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
24 months
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).
24 months
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).
24 months
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).
24 months
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.
24 months
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.
24 months
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.
24 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ascidian Therapeutics, Inc

Investigators

  • Study Director: Alia Rashid, Ascidian Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 11, 2024

Primary Completion (Estimated)

August 1, 2030

Study Completion (Estimated)

December 1, 2030

Study Registration Dates

First Submitted

May 31, 2024

First Submitted That Met QC Criteria

June 14, 2024

First Posted (Actual)

June 21, 2024

Study Record Updates

Last Update Posted (Actual)

December 2, 2025

Last Update Submitted That Met QC Criteria

November 25, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ACDN-01-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe