RECRUITING

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

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Conditions

Stargardt DiseaseCone Rod DystrophyJuvenile Macular DegenerationStargardt Disease 1ABCA4ABCA4-related retinopathyStargardt macular dystrophyGene editingRNAGene TherapyExon editingIRDInherited retinal diseaseInherited retinal dystrophyInherited retinal degeneration

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2024-06-11
Study Completion:2030-12-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06467344

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Presence of mutations in the ABCA4 gene
  2. * ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
  3. * Area of atrophy located in the macula of the study eye
  4. * BCVA of 20/80 (0.6 logMAR) or worse
  1. * The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy
  2. * Retinal disease other than ABCA4-related retinopathy
  3. * 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.

Contacts and Locations

Study Contact

Associate Director, Clinical Operations
CONTACT
207-573-0412
researchtrials@ascidian-tx.com

Principal Investigator

Alia Rashid
STUDY_DIRECTOR
Ascidian Therapeutics

Study Locations (Sites)

University of San Francisco
San Francisco, California, 94158
United States
Vitreo Retinal Associates
Gainesville, Florida, 32607
United States
Bascom Palmer Eye Institute
Miami, Florida, 33136
United States
Wilmer Eye Institute at John Hopkins
Baltimore, Maryland, 21218
United States
Massachusetts Eye and Ear
Boston, Massachusetts, 02114
United States
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, 48105
United States
Cincinnati Eye Institute
Cincinnati, Ohio, 45245
United States
Casey Eye Institute OHSU
Portland, Oregon, 97239
United States
Retina Foundation of the Southwest
Dallas, Texas, 75382
United States
Retina Consultants of Texas
Houston, Texas, 77401
United States

Collaborators and Investigators

Sponsor: Ascidian Therapeutics, Inc

  • Alia Rashid, STUDY_DIRECTOR, Ascidian Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-06-11
Study Completion Date2030-12-01

Study Record Updates

Study Start Date2024-06-11
Study Completion Date2030-12-01

Terms related to this study

Keywords Provided by Researchers

  • ABCA4
  • ABCA4-related retinopathy
  • Stargardt Disease
  • Stargardt macular dystrophy
  • Cone rod dystrophy
  • Gene editing
  • RNA
  • Gene Therapy
  • Exon editing
  • IRD
  • Inherited retinal disease
  • Inherited retinal dystrophy
  • Inherited retinal degeneration

Additional Relevant MeSH Terms

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