Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorAscidian Therapeutics, Inc
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
ACDN-01
DrugACDN-01 is an AAV-based vector carrying a DNA construct encoding for an ABCA4 RNA exon editor. One time administration is via subretinal injection.
Treatment groups
15 Participants
are divided into 3 treatment groupsGroup A: Low dose of ACDN-01Experimental treatment 1 intervention
Group B: Mid-dose of ACDN-01Experimental treatment 1 intervention
Group C: High dose of ACDN-01Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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.
Time frame
12 monthsSecondary outcomes
1
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.
Time frame
12 months2
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.
Time frame
24 months3
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.
Time frame
24 monthsOther outcomes
1
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.
Time frame
24 months2
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).
Time frame
24 months3
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).
Time frame
24 months4
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).
Time frame
24 monthsSponsors and contacts
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Source ClinicalTrials.gov