NCT06467344

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Ascidian Therapeutics, Inc · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Stargardt Disease
• Cone Rod Dystrophy
• Juvenile Macular Degeneration
• Stargardt Disease 1

Interventions

Type	Name	Description
DRUG	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.

Timeline

• Start date: 2024-06-11
• Primary completion: 2030-08-01
• Completion: 2030-12-01
• First posted: 2024-06-21
• Last updated: 2025-12-02

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study