Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)

NCT06445322 | Recruiting

• 1 other identifier: ACDN-01-000
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 8

Brief Summary

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.

Conditions

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

Keywords

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

Outcome Measures

Primary Outcomes (7)

• Confirm mutations in the ABCA4 gene

  Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.

  12 months

• Confirm the absence of pathogenic mutations in genes known to cause retinal disease other than ABCA4-related retinopathy

  Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.

  12 months

• Measure BCVA and LLVA

  Measure best corrected visual acuity and low luminance visual acuity

  12 months

• Measure the area of retinal atrophy

  Using FAF imaging

  12 months

• Measure baseline retinal structure

  Using OCT (SD-OCT)

  12 months

• Historical FAF or OCT images

  Confirm historical timepoint images

  4 years

• Historical BCVA/LLVA measurements

  Collect past measurements

  4 years

Study Arms (1)

Prescreening Group

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.

Diagnostic Test: Prescreening Assessments

Interventions

Prescreening Assessments DIAGNOSTIC_TEST

Various genetic and visual assessments.

Prescreening Group

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Males and females with a diagnosis of ABCA4-related retinopathy will be invited to give informed consent prior to prescreening procedures. After appropriate informed consent/assent has been obtained, potential participants will then undergo the assessments to determine eligibility for ACDN-01 clinical trials.

You may qualify if:

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

You may not qualify 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.

Sponsors & Collaborators

• Ascidian Therapeutics, Inc: lead

Study Sites (8)

University of San Francisco

San Francisco, California, 94158, United States

RECRUITING

Vitreo Retinal Associates

Gainesville, Florida, 32607, United States

RECRUITING

Wilmer Eye Institute at John Hopkins

Baltimore, Maryland, 21218, United States

RECRUITING

Massachusetts Eye and Ear

Boston, Massachusetts, 02114, United States

RECRUITING

University of Michigan Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

RECRUITING

Cincinnati Eye Institute

Cincinnati, Ohio, 45245, United States

RECRUITING

Retina Foundation of Texas

Dallas, Texas, 75382, United States

RECRUITING

Retina Consultants of Texas

Houston, Texas, 77401, United States

RECRUITING

MeSH Terms

Conditions

Stargardt Disease; Cone-Rod Dystrophies

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Retinal Dystrophies

Study Officials

• Alia Rashid

  Ascidian Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Associate Director, Clinical Operations

CONTACT

207-573-0412; researchtrials@ascidian-tx.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 31, 2024
• First Posted: June 6, 2024
• Study Start: June 20, 2024
• Primary Completion (Estimated): August 31, 2027
• Study Completion (Estimated): August 31, 2030
• Last Updated: March 11, 2026

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)