Prescreening Study to Identify Potential Participants for ACDN-01 Clinical Trials

Ascidian Therapeutics, Inc

Status: Enrolling

Conditions: Cone Rod Dystrophy; Stargardt Disease; Stargardt Disease 1; Juvenile Macular Degeneration

Treatments: Diagnostic Test: Prescreening Assessments

Study type: Observational

Funder types: Industry

Identifiers

NCT06445322

ACDN-01-000

Details and patient eligibility

About

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.

Full description

The prescreening process will be used to help determine the initial eligibility and interest of potential participants in ACDN-01 clinical trials by conducting assessments of key eligibility criteria before the clinical trial screening procedures are performed.

Enrollment: 50 estimated patients

Sex: All

Ages: 5+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Presence of mutations in the ABCA4 gene
• ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)

Key Exclusion Criteria:

• 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

50 participants in 1 patient group

Prescreening Group

Description:

The prescreening study consists of genetic and visual assessments and will require at least 1 onsite visit. All clinical assessments performed are for the purpose of determining research eligibility for ACDN-01 clinical trials.

Treatment:

Diagnostic Test: Prescreening Assessments