Stargardt DiseaseCone Rod DystrophyJuvenile Macular DegenerationStargardt Disease 1
Investigational drug(s) / intervention(s)
ACDN-01
ACDN-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.
Study 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.
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
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 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.
Primary outcome measure(s)
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.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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