Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
NCT ID: NCT06467344
Last Updated: 2025-12-02
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
15 participants
INTERVENTIONAL
2024-06-11
2030-12-01
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Low dose of ACDN-01
One time low dose of ACDN-01.
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.
Mid-dose of ACDN-01
One time mid-dose of ACDN-01.
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.
High dose of ACDN-01
One time high dose of ACDN-01.
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.
Interventions
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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.
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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.
12 Years
ALL
No
Sponsors
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Ascidian Therapeutics, Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Alia Rashid
Role: STUDY_DIRECTOR
Ascidian Therapeutics
Locations
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University of San Francisco
San Francisco, California, United States
Vitreo Retinal Associates
Gainesville, Florida, United States
Bascom Palmer Eye Institute
Miami, Florida, United States
Wilmer Eye Institute at John Hopkins
Baltimore, Maryland, United States
Massachusetts Eye and Ear
Boston, Massachusetts, United States
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, United States
Cincinnati Eye Institute
Cincinnati, Ohio, United States
Casey Eye Institute OHSU
Portland, Oregon, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
Retina Consultants of Texas
Houston, Texas, United States
Countries
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Central Contacts
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References
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Doi A, Delaney C, Tanner D, Burkhart K, Bell RD. RNA exon editing: Splicing the way to treat human diseases. Mol Ther Nucleic Acids. 2024 Aug 16;35(3):102311. doi: 10.1016/j.omtn.2024.102311. eCollection 2024 Sep 10.
Other Identifiers
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ACDN-01-001
Identifier Type: -
Identifier Source: org_study_id
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