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

Last updated: May 12, 2025
Sponsor: Ascidian Therapeutics, Inc
Overall Status: Active - Recruiting

Phase

1/2

Condition

Retina

Macular Degeneration

Myopic Macular Degeneration

Treatment

ACDN-01

Clinical Study ID

NCT06467344
ACDN-01-001
  • Ages > 18
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

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 20/80 (0.6 logMAR) or worse

Exclusion

Key Exclusion Criteria:

  • The presence of pathogenic or likely pathogenic mutations in other genes known tocause 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 findingthat may in the opinion of the principal investigator and sponsor preclude adherenceto the scheduled study visits, safe participation in the study, or affect theresults of the study.

Study Design

Total Participants: 13
Treatment Group(s): 1
Primary Treatment: ACDN-01
Phase: 1/2
Study Start date:
June 11, 2024
Estimated Completion Date:
December 01, 2030

Study 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.

Connect with a study center

  • University of San Francisco

    San Francisco, California 94158
    United States

    Active - Recruiting

  • Vitreo Retinal Associates

    Gainesville, Florida 32607
    United States

    Active - Recruiting

  • Bascom Palmer Eye Institute

    Miami, Florida 33136
    United States

    Active - Recruiting

  • Wilmer Eye Institute at John Hopkins

    Baltimore, Maryland 21218
    United States

    Active - Recruiting

  • Massachusetts Eye and Ear

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • University of Michigan Kellogg Eye Center

    Ann Arbor, Michigan 48105
    United States

    Active - Recruiting

  • Cincinnati Eye Institute

    Cincinnati, Ohio 45245
    United States

    Active - Recruiting

  • Casey Eye Institute OHSU

    Portland, Oregon 97239
    United States

    Active - Recruiting

  • Retina Foundation of the Southwest

    Dallas, Texas 75382
    United States

    Active - Recruiting

  • Retina Consultants of Texas

    Houston, Texas 77401
    United States

    Active - Recruiting

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