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

Stargardt Disease Clinical Trial

Official title:

Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06445322
• Other study ID #: ACDN-01-000
• Status: Recruiting
• Start date: June 20, 2024
• Est. completion date: August 31, 2030

Study information

• Verified date: June 2024
• Source: Ascidian Therapeutics, Inc
• Contact: Associate Director, Clinical Operations
• Phone: 207-573-0412
• Email: researchtrials[@]ascidian-tx.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: August 31, 2030
• Est. primary completion date: August 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

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

Intervention

Diagnostic Test:
• Prescreening Assessments
Various genetic and visual assessments.

Locations

Country	Name	City	State
United States	Cincinnati Eye Institute	Cincinnati	Ohio
United States	Retina Foundation of Texas	Dallas	Texas
United States	Vitreo Retinal Associates	Gainesville	Florida
United States	Retina Consultants of Texas	Houston	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Ascidian Therapeutics, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Confirm mutations in the ABCA4 gene	Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.	12 months
Primary	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
Primary	Measure BCVA and LLVA	Measure best corrected visual acuity and low luminance visual acuity	12 months
Primary	Measure the area of retinal atrophy	Using FAF imaging	12 months
Primary	Measure baseline retinal structure	Using OCT (SD-OCT)	12 months
Primary	Historical FAF or OCT images	Confirm historical timepoint images	4 years
Primary	Historical BCVA/LLVA measurements	Collect past measurements	4 years