Health TrialsbyHealthPanel

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

ConditionsStargardt DiseaseStargardt Disease 1Cone Rod DystrophyJuvenile Macular Degeneration
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age5+
SponsorAscidian Therapeutics, Inc

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

This trial does not accept healthy volunteers

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 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.

Trial design

Design model

Cohort

Time perspective

Cross-sectional

Treatments tested in this trial

  • Prescreening Assessments

    Diagnostic test

    Various genetic and visual assessments.

Treatment groups

50 Participants
are divided into 1 treatment group
Group A: Prescreening Group1 intervention

Trial outcomes

Primary outcomes

1

Confirm mutations in the ABCA4 gene

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

Time frame
12 months
2

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.

Time frame
12 months
3

Measure BCVA and LLVA

Measure best corrected visual acuity and low luminance visual acuity

Time frame
12 months
4

Measure the area of retinal atrophy

Using FAF imaging

Time frame
12 months

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Ascidian Therapeutics, Inc

Lead sponsor

Source ClinicalTrials.gov
Apply now