Clinical trial NCT06445322: Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials
Official title: Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Stargardt Disease
Stargardt Disease 1
Cone Rod Dystrophy
Juvenile Macular Degeneration
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
Lead sponsor: Ascidian Therapeutics, Inc
Sponsor class: INDUSTRY
First posted: 2024-06-06
Enrollment: 50
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
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.

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.

Detailed 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.

Eligibility 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.

Primary outcomes:
measure: Confirm mutations in the ABCA4 gene
timeFrame: 12 months
measure: Confirm the absence of pathogenic mutations in genes known to cause retinal disease other than ABCA4-related retinopathy
timeFrame: 12 months
measure: Measure BCVA and LLVA
timeFrame: 12 months
measure: Measure the area of retinal atrophy
timeFrame: 12 months
measure: Measure baseline retinal structure
timeFrame: 12 months
measure: Historical FAF or OCT images
timeFrame: 4 years
measure: Historical BCVA/LLVA measurements
timeFrame: 4 years
Secondary outcomes:
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of San Francisco
state: California
city: Gainesville
country: United States
facility: Vitreo Retinal Associates
state: Florida
city: Baltimore
country: United States
facility: Wilmer Eye Institute at John Hopkins
state: Maryland
city: Boston
country: United States
facility: Massachusetts Eye and Ear
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Kellogg Eye Center
state: Michigan
city: Cincinnati
country: United States
facility: Cincinnati Eye Institute
state: Ohio
city: Dallas
country: United States
facility: Retina Foundation of Texas
state: Texas
city: Houston
country: United States
facility: Retina Consultants of Texas
state: Texas
Study officials:
affiliation: Ascidian Therapeutics
name: Alia Rashid
role: STUDY_DIRECTOR
References: