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

a study on Stargardt Disease Cone-Rod Dystrophy Macular Degeneration

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around
Principal Investigator
by Jacque Duncan
Headshot of Jacque Duncan
Jacque Duncan

Description

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.

Official Title

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

Details

This is an open-label, single ascending dose study of ACDN-01 in adult participants with ABCA4-related retinopathy. The study is designed to evaluate the safety, tolerability, and evidence of biological effect of SAD levels (low, medium, and high) of ACDN-01 when delivered subretinally.

Participants will be followed on study for 2 years for the primary safety and preliminary efficacy endpoints, after which they will continue in the study in a 3-year long-term follow-up period, for a total study duration of 5 years.

Keywords

Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1, ABCA4, ABCA4-related retinopathy, Stargardt macular dystrophy, Gene editing, RNA, Gene Therapy, Exon editing, IRD, Inherited retinal disease, Inherited retinal dystrophy, Inherited retinal degeneration, Macular Degeneration, Retinal Diseases, Cone-Rod Dystrophies, ACDN-01

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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 20/80 (0.6 logMAR) or worse

You CAN'T join 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.

Locations

  • University of San Francisco accepting new patients
    San Francisco California 94158 United States
  • Casey Eye Institute OHSU accepting new patients
    Portland Oregon 97239 United States

Lead Scientist at UCSF

  • Jacque Duncan
    Retinitis pigmentosa (RP) affects about 1 in 3,500 people worldwide. Age-related macular degeneration (AMD) affects as many as 1 in 4 people by the age of 75 and is the leading cause of blindness in people over age 50 in the United States. Both RP and AMD have a hereditary basis, and currently, there is no cure for either of these types of hereditary retinal degeneration.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Ascidian Therapeutics, Inc
ID
NCT06467344
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 13 study participants
Last Updated
Contact us about this trial