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

Description

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

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Stargardt Disease
Intervention / Treatment

-

Contacts and Locations

San Francisco

University of San Francisco, San Francisco, California, United States, 94158

Gainesville

Vitreo Retinal Associates, Gainesville, Florida, United States, 32607

Miami

Bascom Palmer Eye Institute, Miami, Florida, United States, 33136

Baltimore

Wilmer Eye Institute at John Hopkins, Baltimore, Maryland, United States, 21218

Boston

Massachusetts Eye and Ear, Boston, Massachusetts, United States, 02114

Ann Arbor

University of Michigan Kellogg Eye Center, Ann Arbor, Michigan, United States, 48105

Cincinnati

Cincinnati Eye Institute, Cincinnati, Ohio, United States, 45245

Portland

Casey Eye Institute OHSU, Portland, Oregon, United States, 97239

Dallas

Retina Foundation of the Southwest, Dallas, Texas, United States, 75382

Houston

Retina Consultants of Texas, Houston, Texas, United States, 77401

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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.

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Ascidian Therapeutics, Inc,

Alia Rashid, STUDY_DIRECTOR, Ascidian Therapeutics

Study Record Dates

2030-12-01