Recruiting

ACDN-01

Sponsor:

Ascidian Therapeutics, Inc

Code:

NCT06445322

Conditions

Stargardt Disease

Stargardt Disease 1

Cone Rod Dystrophy

Juvenile Macular Degeneration

Eligibility Criteria

Sex: All

Age: 5+

Healthy Volunteers: Not accepted

Interventions

Prescreening Assessments

Study Details

Brief summary:

This is an observational prescreening study. Individuals who are eligible for prescreening will undergo testing procedures that may be used to determine eligibility in ACDN-01 clinical trials.

Conditions

Stargardt Disease

Stargardt Disease 1

Cone Rod Dystrophy

Juvenile Macular Degeneration

Study ID

NCT06445322

Start date

Jun 20, 2024

Status verified date

May, 2025

Completion date

Aug 31, 2030

Anticipated

Primary completion date

Aug 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 5+

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)

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

50 participants

Anticipated

Interventions and Outcome Measures

Arms

Prescreening Group

The prescreening study consists of genetic and visual assessments and will require at least 1 onsite visit. All clinical assessments performed are for the purpose of determining research eligibility for ACDN-01 clinical trials.

Interventions

Prescreening Assessments

Various genetic and visual assessments.

Primary outcome measure

  • Confirm mutations in the ABCA4 gene [ Time Frame: 12 months ]
  • Confirm the absence of pathogenic mutations in genes known to cause retinal disease other than ABCA4-related retinopathy [ Time Frame: 12 months ]
  • Measure BCVA and LLVA [ Time Frame: 12 months ]
  • Measure the area of retinal atrophy [ Time Frame: 12 months ]
  • Measure baseline retinal structure [ Time Frame: 12 months ]
  • Historical FAF or OCT images [ Time Frame: 4 years ]
  • Historical BCVA/LLVA measurements [ Time Frame: 4 years ]

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

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

Retina Foundation of Texas

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

Mar 11, 2026

Last verified

May, 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 2026-03-11.