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
Status: Recruiting
Location: See all (10) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Healthy Volunteers: f
View:
• 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
Locations
United States
California
University of San Francisco
RECRUITING
San Francisco
Florida
Vitreo Retinal Associates
RECRUITING
Gainesville
Bascom Palmer Eye Institute
RECRUITING
Miami
Massachusetts
Massachusetts Eye and Ear
RECRUITING
Boston
Maryland
Wilmer Eye Institute at John Hopkins
RECRUITING
Baltimore
Michigan
University of Michigan Kellogg Eye Center
RECRUITING
Ann Arbor
Ohio
Cincinnati Eye Institute
RECRUITING
Cincinnati
Oregon
Casey Eye Institute OHSU
RECRUITING
Portland
Texas
Retina Foundation of the Southwest
RECRUITING
Dallas
Retina Consultants of Texas
RECRUITING
Houston
Contact Information
Primary
Associate Director, Clinical Operations
researchtrials@ascidian-tx.com
207-573-0412
Time Frame
Start Date: 2024-06-11
Estimated Completion Date: 2030-12-01
Participants
Target number of participants: 15
Treatments
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.
Related Therapeutic Areas
Sponsors
Leads: Ascidian Therapeutics, Inc