Recruiting
Phase 1
Phase 2

ACDN-01

Sponsor:

Ascidian Therapeutics, Inc

Code:

NCT06467344

Conditions

Stargardt Disease

Cone Rod Dystrophy

Juvenile Macular Degeneration

Stargardt Disease 1

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

Interventions

ACDN-01

Study Details

Brief 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.

Conditions

Stargardt Disease

Cone Rod Dystrophy

Juvenile Macular Degeneration

Stargardt Disease 1

Study ID

NCT06467344

Start date

Jun 11, 2024

Status verified date

Nov, 2025

Completion date

Dec 1, 2030

Anticipated

Primary completion date

Aug 1, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

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 Design

Enrollment

15 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Low dose of ACDN-01

One time low dose of ACDN-01.

experimental: Mid-dose of ACDN-01

One time mid-dose of ACDN-01.

experimental: High dose of ACDN-01

One time high dose of ACDN-01.

Interventions

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.

Primary outcome measure

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

Central Contacts and Locations

Central contacts

Associate Director, Clinical Operations

207-573-0412researchtrials@ascidian-tx.com

Locations

University of San Francisco

Recruiting

San Francisco, California, United States, 94158

Vitreo Retinal Associates

Recruiting

Gainesville, Florida, United States, 32607

Bascom Palmer Eye Institute

Recruiting

Miami, Florida, United States, 33136

Wilmer Eye Institute at John Hopkins

Recruiting

Baltimore, Maryland, United States, 21218

Massachusetts Eye and Ear

Recruiting

Boston, Massachusetts, United States, 02114

University of Michigan Kellogg Eye Center

Recruiting

Ann Arbor, Michigan, United States, 48105

Cincinnati Eye Institute

Recruiting

Cincinnati, Ohio, United States, 45245

Casey Eye Institute OHSU

Recruiting

Portland, Oregon, United States, 97239

Retina Foundation of the Southwest

Recruiting

Dallas, Texas, United States, 75382

Retina Consultants of Texas

Recruiting

Houston, Texas, United States, 77401

More Information

Sponsor

Ascidian Therapeutics, Inc

Last update posted

Dec 2, 2025

Last verified

Nov, 2025

Keywords

  • ABCA4
  • ABCA4-related retinopathy
  • Stargardt Disease
  • Stargardt macular dystrophy
  • Cone rod dystrophy
  • Gene editing
  • RNA
  • Gene Therapy
  • Exon editing
  • IRD
  • Inherited retinal disease
  • Inherited retinal dystrophy
  • Inherited retinal degeneration

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Ascidian Therapeutics, Inc on 2025-12-02.