Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
- Conditions
- Stargardt DiseaseCone Rod DystrophyJuvenile Macular DegenerationStargardt Disease 1
- Interventions
- Registration Number
- NCT06467344
- Lead Sponsor
- Ascidian Therapeutics, Inc
- Brief 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 13
- 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
Key
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Low dose of ACDN-01 ACDN-01 One time low dose of ACDN-01. Mid-dose of ACDN-01 ACDN-01 One time mid-dose of ACDN-01. High dose of ACDN-01 ACDN-01 One time high dose of ACDN-01.
- Primary Outcome Measures
Name Time Method Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events. 12 months To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy.
- Secondary Outcome Measures
Name Time Method Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data. 12 months To determine maximum tolerated dose (MTD) for subsequent clinical evaluation.
To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF). 24 months To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in the area of macular atrophy in mm2 as measured on FAF over 24 months.
To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT). 24 months To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in change in thickness in retinal layers as measured on OCT, over 24 months.
Trial Locations
- Locations (10)
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