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

Phase 2RecruitingNCT06467344

Ascidian Therapeutics, Inc15 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Stargardt Disease Cone Rod Dystrophy Juvenile Macular Degeneration Stargardt Disease 1

Interventions

ACDN-01DRUG

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.

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

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.

Locations (10)

University of San Francisco

San Francisco, California, United States

RECRUITING

Vitreo Retinal Associates

Gainesville, Florida, United States

RECRUITING

Outcomes

Primary Outcomes

Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.

To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy.

Time frame: 12 months

Secondary Outcomes

Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.

To determine maximum tolerated dose (MTD) for subsequent clinical evaluation.

Time frame: 12 months

To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).

To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in the area of macular atrophy in mm2 as measured on FAF over 24 months.

Time frame: 24 months

To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).

To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in change in thickness in retinal layers as measured on OCT, over 24 months.

Time frame: 24 months

Central Contacts

Associate Director, Clinical Operations

CONTACT

207-573-0412 researchtrials@ascidian-tx.com

Data from ClinicalTrials.gov

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