About this trial

This is an observational prescreening study. Individuals who are eligible for prescreening will undergo testing procedures that may be used to determine eligibility in ACDN-01 clinical trials.

Eligibility criteria

Qualifiers

Presence of mutations in the ABCA4 gene

ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)

Disqualifiers

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.

Trial design

Treatments tested in this trial

  • Prescreening Assessments

Treatment groups

50 Participants
are divided into 1 treatment group

Locations

8
California
University of San Francisco94158, San Francisco United States
Florida
Vitreo Retinal Associates32607, Gainesville United States
Maryland
Wilmer Eye Institute at John Hopkins21218, Baltimore United States
Massachusetts
Massachusetts Eye and Ear02114, Boston United States

Sponsors and collaborators

Ascidian Therapeutics, Inc

Lead sponsor