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

Part of paid clinical trials in San Francisco, California.

Sponsor
Ascidian Therapeutics, Inc
Study ID
NCT06467344
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Cone Rod Dystrophy
  • Juvenile Macular Degeneration
  • Stargardt Disease
  • Stargardt Disease 1

Eligibility Criteria

Sex
ALL
Age
12 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • ACDN-01 — DRUG
    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 Details

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.

Key Dates

First listed
Jun 21, 2024
Start date
Jun 11, 2024
Status verified
Nov 2025
Primary completion
Aug 1, 2030
Completion
Dec 1, 2030

Study Design

Enrollment
15 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Low dose of ACDN-01
    One time low dose of ACDN-01.
  • Experimental: Mid-dose of ACDN-01
    One time mid-dose of ACDN-01.
  • Experimental: High dose of ACDN-01
    One time high dose of ACDN-01.

Primary Outcome Measure

Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events. [ Time Frame: 12 months ]

Central Contacts

Locations (10)

FacilityCityStateZIPSite coordinators
University of San FranciscoSan FranciscoCalifornia94158-
Vitreo Retinal AssociatesGainesvilleFlorida32607-
Bascom Palmer Eye InstituteMiamiFlorida33136-
Wilmer Eye Institute at John HopkinsBaltimoreMaryland21218-
Massachusetts Eye and EarBostonMassachusetts02114-
University of Michigan Kellogg Eye CenterAnn ArborMichigan48105-
Cincinnati Eye InstituteCincinnatiOhio45245-
Casey Eye Institute OHSUPortlandOregon97239-
Retina Foundation of the SouthwestDallasTexas75382-
Retina Consultants of TexasHoustonTexas77401-

Find similar trials in San Francisco, CA

By research site
University of San Francisco· San Francisco, CAVitreo Retinal Associates· Gainesville, FLBascom Palmer Eye Institute· Miami, FLWilmer Eye Institute at John Hopkins· Baltimore, MDMassachusetts Eye and Ear· Boston, MAUniversity of Michigan Kellogg Eye Center· Ann Arbor, MI

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