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Clinical Trial Report

Gene Therapy Trial Report

Summary

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


NCTID NCT06467344 (View at clinicaltrials.gov)
Description
Indication Stargardt Disease
Compound Name ACDN-01
Sponsor Ascidian Therapeutics, Inc
Funder Type Industry
Status
Recruiting
Enrollment Count 13

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
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV
Editor Type
Dose 1 Undisclosed low dose
Dose 2 Undisclosed medium dose
Dose 3 Undisclosed high dose
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-05-31
Completion Date 2030-12-01
Last Update 2024-10-15

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 10
Locations United States

Regulatory Information


Has US IND True
Recent Updates Rare Pediatric Disease designation granted 4/25/24

Resources/Links