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Clinical Trials in the USA / NCT06467344
Recruiting Phase 1/2

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

NCT06467344 · tracked via the Priya Life Science USA tracker
Sponsor
Ascidian Therapeutics, Inc
Phase
Phase 1/2
Started
2024-06-11
Last updated
2026-10-07

Condition(s) studied

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.

Trial sites (11)

FacilityCityRegionStatus
University of San Francisco San Francisco California Terminated
Vitreo Retinal Associates Gainesville Florida Recruiting
Bascom Palmer Eye Institute Miami Florida Recruiting
Wilmer Eye Institute at John Hopkins Baltimore Maryland Terminated
Massachusetts Eye and Ear Boston Massachusetts Recruiting
University of Michigan Kellogg Eye Center Ann Arbor Michigan Recruiting
Cincinnati Eye Institute Cincinnati Ohio Recruiting
Casey Eye Institute OHSU Portland Oregon Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
Retina Foundation of the Southwest Dallas Texas Recruiting
Retina Consultants of Texas Houston Texas Recruiting

More Ascidian Therapeutics, Inc trials in the USA

Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH) Recruiting

Other trials for the same condition

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1) Recruiting
An Observational Study in Children and Adults With Stargardt Disease Active, not recruiting
Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration Recruiting
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene Recruiting
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease Recruiting
Official registry record

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.

View NCT06467344 on ClinicalTrials.gov ↗ ← All trials in the USA