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NCT06467344PHASE1, PHASE2Recruiting

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

Ascidian Therapeutics, Inc

Start Date

6/11/2024

Completion Date

12/1/2030

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.

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.

Eligibility Criteria

Age Range: 12 years to No maximum

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.

Interventions

DRUG

ACDN-01

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Conditions

Stargardt DiseaseCone Rod DystrophyJuvenile Macular DegenerationStargardt Disease 1

Locations

University of San Francisco

San Francisco, California 94158

United States

Vitreo Retinal Associates

Gainesville, Florida 32607

United States

Bascom Palmer Eye Institute

Miami, Florida 33136

United States

Wilmer Eye Institute at John Hopkins

Baltimore, Maryland 21218

United States

Massachusetts Eye and Ear

Boston, Massachusetts 02114

United States

University of Michigan Kellogg Eye Center

Ann Arbor, Michigan 48105

United States

Cincinnati Eye Institute

Cincinnati, Ohio 45245

United States

Casey Eye Institute OHSU

Portland, Oregon 97239

United States

Retina Foundation of the Southwest

Dallas, Texas 75382

United States

Retina Consultants of Texas

Houston, Texas 77401

United States