RecruitingNot Applicable

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

United States50 participantsStarted 2024-06-20

Plain-language 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.

Who can participate

Age range

5 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Confirm mutations in the ABCA4 gene

Timeframe: 12 months

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

Timeframe: 12 months

Measure BCVA and LLVA

Timeframe: 12 months

Measure the area of retinal atrophy

Timeframe: 12 months

Measure baseline retinal structure

Timeframe: 12 months

Historical FAF or OCT images

Timeframe: 4 years

Historical BCVA/LLVA measurements

Timeframe: 4 years

Trial details

NCT IDNCT06445322

SponsorAscidian Therapeutics, Inc

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2027-08-31

Contact for this trial

Associate Director, Clinical Operations

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

View on ClinicalTrials.gov More Stargardt Disease trials

Who can participate

Age range

5 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Confirm mutations in the ABCA4 gene

Timeframe: 12 months

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

Timeframe: 12 months

Measure BCVA and LLVA

Timeframe: 12 months

Measure the area of retinal atrophy

Timeframe: 12 months

Measure baseline retinal structure

Timeframe: 12 months

Historical FAF or OCT images

Timeframe: 4 years

Historical BCVA/LLVA measurements

Timeframe: 4 years