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NCT Number: NCT06467344

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

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.

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Key information

Conditions

Stargardt Disease Cone Rod Dystrophy Cone-Rod Dystrophies Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Juvenile Macular Degeneration Macular Degeneration Retinal Degeneration Retinal Diseases Retinal Dystrophies Stargardt Disease 1

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University of San Francisco, San Francisco, California, United States

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About this study

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 can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 and study plan

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.

Primary outcomes

  1. 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 outcomes

  1. 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.

  2. 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.

  3. 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 outcomes

  1. 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.

  2. 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).

  3. 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).

  4. 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).

  5. 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.

  6. 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.

  7. 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Associate Director, Clinical Operations

CONTACT

[email protected]

207-573-0412

Sponsors and collaborators

Lead sponsor

Ascidian Therapeutics, Inc

Industry

Registry information

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

Important dates

Study start
2024
Primary completion
2030
Study completion
2030
First posted
Jun 21, 2024
Registry last updated
Dec 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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