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
Qualifiers
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
Treatments tested in this trial
- ACDN-01
Treatment groups
15 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov