Stargardt DiseaseNCT06445322
Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)
An observational study of Stargardt Disease and Stargardt Disease 1, run by Ascidian Therapeutics, Inc at 9 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Stargardt Disease, Stargardt Disease 1, Cone Rod Dystrophy
- What is being tested?
- Prescreening Assessments
- How many people
- 50 participants
- Where
- 9 sitesUnited States
- Study dates
- 20 Jun 2024to 31 Aug 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- Presence of mutations in the ABCA4 gene
- ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
Who cannot join
- 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 locations and contacts
9 sites in 1 country. Contact the study team or a site near you to ask about taking part.
9 sites in 1 country
United States9 sites, 7 recruiting
- Recruiting
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, 48105
- Terminated
Wilmer Eye Institute at John Hopkins
Baltimore, Maryland, 21218
- Recruiting
Massachusetts Eye and Ear
Boston, Massachusetts, 02114
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
- Recruiting
Cincinnati Eye Institute
Cincinnati, Ohio, 45245
- Recruiting
Retina Foundation of Texas
Dallas, Texas, 75382
- Recruiting
Vitreo Retinal Associates
Gainesville, Florida, 32607
- Recruiting
Retina Consultants of Texas
Houston, Texas, 77401
- Terminated
University of San Francisco
San Francisco, California, 94158
About this study
Official title: Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials
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.
Read the 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.
- Being tested
- Prescreening Assessments
- 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
- Medical subject headings
- Stargardt Disease, Cone-Rod Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Prescreening AssessmentsDiagnostic test
Various genetic and visual assessments.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Diagnostic Test: Prescreening Assessments |
What is being measured
The primary outcome is the main question the study is designed to answer.
Confirm mutations in the ABCA4 gene
Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.
Measured: 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.
Measured: 12 months
Measure BCVA and LLVA
Measure best corrected visual acuity and low luminance visual acuity
Measured: 12 months
Measure the area of retinal atrophy
Using FAF imaging
Measured: 12 months
Measure baseline retinal structure
Using OCT (SD-OCT)
Measured: 12 months
Historical FAF or OCT images
Confirm historical timepoint images
Measured: 4 years
Historical BCVA/LLVA measurements
Collect past measurements
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2024
- Study start20 Jun 2024
- Primary completion31 Aug 2027 (estimated)
- Study completion31 Aug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ascidian Therapeutics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Murray Abramson, MD, PhD, Study director, Ascidian Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06445322
- Sponsor's protocol ID
- ACDN-01-000
- Status last verified
- Oct 2026
- First submitted
- 31 May 2024
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials
- ClinicalTrials.gov ID
- NCT06445322
- Acronym / sponsor ID
- ACDN-01-000
- Keywords
- ABCA4, ABCA4-related retinopathy, Stargardt Disease, Stargardt macular dystrophy, Cone rod dystrophy, Gene editing, RNA, Gene Therapy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Ascidian Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- 20 Jun 2024 (actual)
- Primary completion
- 31 Aug 2027 (estimated)
- Study completion
- 31 Aug 2030 (estimated)
- First posted
- 6 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Prescreening Group | – | Diagnostic Test: Prescreening AssessmentsThe 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. |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- Presence of mutations in the ABCA4 gene
- ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Prescreening AssessmentsVarious genetic and visual assessments. | Diagnostic test | Prescreening Group |
Primary outcome
Confirm mutations in the ABCA4 gene
Time frame: 12 months
Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.
Confirm the absence of pathogenic mutations in genes known to cause retinal disease other than ABCA4-related retinopathy
Time frame: 12 months
Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.
Measure BCVA and LLVA
Time frame: 12 months
Measure best corrected visual acuity and low luminance visual acuity
Measure the area of retinal atrophy
Time frame: 12 months
Using FAF imaging
Measure baseline retinal structure
Time frame: 12 months
Using OCT (SD-OCT)
Historical FAF or OCT images
Time frame: 4 years
Confirm historical timepoint images
Historical BCVA/LLVA measurements
Time frame: 4 years
Collect past measurements
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Stargardt Disease
RecruitingObservational study
Rod and Cone Mediated Function in Retinal Disease
Retinal Degeneration, Retinitis Pigmentosa
RecruitingObservational study
Genotype-Phenotype Study of Patients With Plaquenil -Induced Retinal Toxicity, With Evaluation of the ABCA4 Gene
Genotype, Retinal Disease
RecruitingPhase 1
Phase 1 Study of C.001 in Retinal Degeneration
Geographic Atrophy, Stargardt Disease
RecruitingPhase 1
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
Stargardt Disease, Stargardt Macular Dystrophy
Active, not recruitingPhase 1/2
Oral Metformin for Treatment of ABCA4 Retinopathy
ABCA4 Retinopathy, Stargardt Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Murray Abramson, MD, PhD study director
Ascidian Therapeutics
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Stargardt Disease and Stargardt Disease 1, run by Ascidian Therapeutics, Inc at 9 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Senior Clinical Trial Manager (207-573-0412, researchtrials@ascidian-tx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Confirm mutations in the ABCA4 gene (time frame: 12 months). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2026.