RecruitingPhase 1Phase 2NCT06467344

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

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

Sponsor

Ascidian Therapeutics, Inc

Enrollment

15 participants

Start Date

Jun 11, 2024

Study Type

INTERVENTIONAL

Conditions

Stargardt Disease

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

Min Age: 12 Years

Inclusion Criteria4

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

Exclusion Criteria3

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.

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