Study to Evaluate ACDN-01 in ABCA4-related Retinopathy (STELLAR)
NCT Number: NCT06467344
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE1|PHASE2
Conditions: Stargardt Disease|Cone Rod Dystrophy|Juvenile Macular Degeneration|Stargardt Disease 1
Interventions: DRUG: ACDN-01
Sponsor: Ascidian Therapeutics, Inc
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 13
Study Design: Allocation: NON_RANDOMIZED|Intervention Model: SEQUENTIAL|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-06-11
Completion Date: 2030-12-01
Locations: Vitreo Retinal Associates, Gainesville, Florida, 32607, United States|Cincinnati Eye Institute, Cincinnati, Ohio, 45245, United States|Retina Foundation of the Southwest, Dallas, Texas, 75382, United States|Retina Consultants of Texas, Houston, Texas, 77401, United States

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.

Primary Outcome Measures: Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events., To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy., 12 months
Secondary Outcome Measures: Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data., To determine maximum tolerated dose (MTD) for subsequent clinical evaluation., 12 months|To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF)., 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., 24 months|To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT)., 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., 24 months
Other Outcome Measures: To assess for evidence of preliminary efficacy of ACDN-01 based on best corrected visual acuity., Best Corrected Visual Acuity is measured by number of ETDRS letters read in each eye compared to baseline., 24 months|To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures., Patient-reported outcome measures will be measured by overall and subscale scores on the Michigan Retinal -Degeneration Questionnaire (MRDQ)., 24 months|To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity., Contrast sensitivity is measured using qCSF tests and will measure number of letters read and change in AUC (area under curve)., 24 months|To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity., Contrast sensitivity is measured using Pelli-Robson tests and will measure number of letters read and change in AUC (area under curve)., 24 months|To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry., Retinal sensitivity will be measured by change in the location of detectable points., 24 months|To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry., Retinal sensitivity will be measured by change in the intensity (dB) of detectable points., 24 months|To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry., Retinal sensitivity will be measured by change in the number of detectable points., 24 months