ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy
Ascidian Therapeutics, Inc
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.
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.
Eligibility
- Age range
- 12+ years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: * 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 Key Exclusion Criteria: * 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 o…
Interventions
- DrugACDN-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.
Locations (10)
- University of San FranciscoSan Francisco, California
- Vitreo Retinal AssociatesGainesville, Florida
- Bascom Palmer Eye InstituteMiami, Florida
- Wilmer Eye Institute at John HopkinsBaltimore, Maryland
- Massachusetts Eye and EarBoston, Massachusetts
- University of Michigan Kellogg Eye CenterAnn Arbor, Michigan