Stargardt DiseasePhase 1/2NCT06467344
Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
A Phase 1/2 study testing ACDN-01 in people with Stargardt Disease and Cone Rod Dystrophy, run by Ascidian Therapeutics, Inc at 11 sites in United States.
Trial summary
This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration
- What is being tested?
- ACDN-01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 11 sitesUnited States
- Study dates
- 11 Jun 2024to 1 Dec 2030
- Phase
- Phase 1/2
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Presence of mutations in the ABCA4 gene
- ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
- Area of atrophy located in the macula of the study eye
- BCVA of 20/50 (0.4 logMAR) or worse
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
11 sites in 1 country. Contact the study team or a site near you to ask about taking part.
11 sites in 1 country
United States11 sites, 9 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
Cincinnati Eye Institute
Cincinnati, Ohio, 45245
- Recruiting
Retina Foundation of the Southwest
Dallas, Texas, 75382
- Recruiting
Vitreo Retinal Associates
Gainesville, Florida, 32607
- Recruiting
Retina Consultants of Texas
Houston, Texas, 77401
- Recruiting
Bascom Palmer Eye Institute
Miami, Florida, 33136
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Recruiting
Casey Eye Institute OHSU
Portland, Oregon, 97239
- Terminated
University of San Francisco
San Francisco, California, 94158
About this study
Official title: ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy
This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.
Read the detailed description
This is an open-label, single ascending dose study of ACDN-01 in adult participants with ABCA4-related retinopathy. The study is designed to evaluate the safety, tolerability, and evidence of biological effect of SAD levels (low, medium, and high) of ACDN-01 when delivered subretinally.
Participants will be followed on study for 2 years for the primary safety and preliminary efficacy endpoints, after which they will continue in the study in a 3-year long-term follow-up period, for a total study duration of 5 years.
- Being tested
- ACDN-01
- 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.
ACDN-01Drug
ACDN-01 is an AAV-based vector carrying a DNA construct encoding for an ABCA4 RNA exon editor. One time administration is via subretinal injection.
| Study group | Role | What they receive |
|---|---|---|
| Low dose of ACDN-01 | Experimental | One time low dose of ACDN-01. Receives: Drug: ACDN-01 |
| Mid-dose of ACDN-01 | Experimental | One time mid-dose of ACDN-01. Receives: Drug: ACDN-01 |
| High dose of ACDN-01 | Experimental | One time high dose of ACDN-01. Receives: Drug: ACDN-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy.
Measured: 12 months
Secondary outcomes (3)
Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.
To determine maximum tolerated dose (MTD) for subsequent clinical evaluation.
Measured: 12 months
To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).
To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in the area of macular atrophy in mm2 as measured on FAF over 24 months.
Measured: 24 months
To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).
To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in change in thickness in retinal layers as measured on OCT, over 24 months.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2024
- Study start11 Jun 2024
- Primary completion1 Aug 2030 (estimated)
- Study completion1 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 15 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
- NCT06467344
- Sponsor's protocol ID
- ACDN-01-001
- Status last verified
- Nov 2025
- First submitted
- 31 May 2024
- Last updated
- 7 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Doi A, Delaney C, Tanner D, Burkhart K, Bell RD. RNA exon editing: Splicing the way to treat human diseases. Mol Ther Nucleic Acids. 2024 Aug 16;35(3):102311. doi: 10.1016/j.omtn.2024.102311. eCollection 2024 Sep 10.
PubMed 39281698
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy
- ClinicalTrials.gov ID
- NCT06467344
- Acronym / sponsor ID
- ACDN-01-001
- Keywords
- ABCA4, ABCA4-related retinopathy, Stargardt Disease, Stargardt macular dystrophy, Cone rod dystrophy, Gene editing, RNA, Gene Therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Ascidian Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- 11 Jun 2024 (actual)
- Primary completion
- 1 Aug 2030 (estimated)
- Study completion
- 1 Dec 2030 (estimated)
- First posted
- 21 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 study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.
Detailed description
This is an open-label, single ascending dose study of ACDN-01 in adult participants with ABCA4-related retinopathy. The study is designed to evaluate the safety, tolerability, and evidence of biological effect of SAD levels (low, medium, and high) of ACDN-01 when delivered subretinally.
Participants will be followed on study for 2 years for the primary safety and preliminary efficacy endpoints, after which they will continue in the study in a 3-year long-term follow-up period, for a total study duration of 5 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low dose of ACDN-01 | Experimental | Drug: ACDN-01One time low dose of ACDN-01. |
| Mid-dose of ACDN-01 | Experimental | Drug: ACDN-01One time mid-dose of ACDN-01. |
| High dose of ACDN-01 | Experimental | Drug: ACDN-01One time high dose of ACDN-01. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Presence of mutations in the ABCA4 gene
- ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
- Area of atrophy located in the macula of the study eye
- BCVA of 20/50 (0.4 logMAR) or worse
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 |
|---|---|---|
| ACDN-01ACDN-01 is an AAV-based vector carrying a DNA construct encoding for an ABCA4 RNA exon editor. One time administration is via subretinal injection. | Drug | High dose of ACDN-01, Low dose of ACDN-01, Mid-dose of ACDN-01 |
Primary outcome
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
Time frame: 12 months
To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy.
3 secondary outcomes
Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.
Time frame: 12 months
To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).
Time frame: 24 months
To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).
Time frame: 24 months
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 7 Oct 2026.
- Derived from this study: PubMed 39281698Doi A, Delaney C, Tanner D, Burkhart K, Bell RD. RNA exon editing: Splicing the way to treat human diseases. Mol Ther Nucleic Acids. 2024 Aug 16;35(3):102311. doi: 10.1016/j.omtn.2024.102311. eCollection 2024 Sep 10.
- Individual participant dataSharing planned: No
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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
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ACDN-01 in people with Stargardt Disease and Cone Rod Dystrophy, run by Ascidian Therapeutics, Inc at 11 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 4 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 7 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 11 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events. (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06467344, which also lists 1 linked publication.
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 7 Oct 2026.