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Gene Therapy Trial Report

Summary

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


NCTID NCT06467344 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration
Disease Ontology Term 🔄 DOID:0050817; DOID:0061241
Compound Name ACDN-01
Sponsor Ascidian Therapeutics, Inc
Funder Type Industry
Recruitment Status
Enrollment Count 15 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant ABCA4 pre-mRNA
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Exon skipping/splice editor
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed medium dose
Dose 3 Undisclosed high dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-05-31
Completion Date 2030-12-01
Last Update 2025-12-02

Participation Criteria


Eligible Age >=12 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 10
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Rare Pediatric Disease Designation
Recent Updates Rare Pediatric Disease designation granted 4/25/24

Resources/Links