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
1 other identifier
interventional
15
1 country
10
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Jun 2024
Longer than P75 for phase_1
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 31, 2024
CompletedStudy Start
First participant enrolled
June 11, 2024
CompletedFirst Posted
Study publicly available on registry
June 21, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2030
December 2, 2025
November 1, 2025
6.1 years
May 31, 2024
November 25, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
12 months
Secondary Outcomes (3)
Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.
12 months
To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).
24 months
To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).
24 months
Other Outcomes (7)
To assess for evidence of preliminary efficacy of ACDN-01 based on best corrected visual acuity.
24 months
To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures.
24 months
To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
24 months
- +4 more other outcomes
Study Arms (3)
Low dose of ACDN-01
EXPERIMENTALOne time low dose of ACDN-01.
Mid-dose of ACDN-01
EXPERIMENTALOne time mid-dose of ACDN-01.
High dose of ACDN-01
EXPERIMENTALOne time high dose of ACDN-01.
Interventions
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 Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (10)
University of San Francisco
San Francisco, California, 94158, United States
Vitreo Retinal Associates
Gainesville, Florida, 32607, United States
Bascom Palmer Eye Institute
Miami, Florida, 33136, United States
Wilmer Eye Institute at John Hopkins
Baltimore, Maryland, 21218, United States
Massachusetts Eye and Ear
Boston, Massachusetts, 02114, United States
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, 48105, United States
Cincinnati Eye Institute
Cincinnati, Ohio, 45245, United States
Casey Eye Institute OHSU
Portland, Oregon, 97239, United States
Retina Foundation of the Southwest
Dallas, Texas, 75382, United States
Retina Consultants of Texas
Houston, Texas, 77401, United States
Related Publications (1)
Doi A, Delaney C, Tanner D, Burkhart K, Bell RD. RNA exon editing: Splicing the way to treat human diseases. Mol Ther Nucleic Acids. 2024 Aug 16;35(3):102311. doi: 10.1016/j.omtn.2024.102311. eCollection 2024 Sep 10.
PMID: 39281698DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Alia Rashid
Ascidian Therapeutics
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 31, 2024
First Posted
June 21, 2024
Study Start
June 11, 2024
Primary Completion (Estimated)
August 1, 2030
Study Completion (Estimated)
December 1, 2030
Last Updated
December 2, 2025
Record last verified: 2025-11
Data Sharing
- IPD Sharing
- Will not share