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โ† Trials
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Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

RecruitingPhase 1, Phase 2Ages 12 Years - anyAllAscidian Therapeutics, Inc
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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.
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What it tests

  • ACDN-01
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Where

11 sites
University of San Francisco
San Francisco, United States
Vitreo Retinal Associates
Gainesville, United States
Bascom Palmer Eye Institute
Miami, United States
Wilmer Eye Institute at John Hopkins
Baltimore, United States
Massachusetts Eye and Ear
Boston, United States
University of Michigan Kellogg Eye Center
Ann Arbor, United States
Cincinnati Eye Institute
Cincinnati, United States
Casey Eye Institute OHSU
Portland, United States

โ€ฆand 3 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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