Stargardt DiseasePhase 1NCT07734064
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
A Phase 1 study testing ASP2020 in people with Stargardt Disease and Stargardt Macular Dystrophy, run by Astellas Institute for Regenerative Medicine at 4 sites in United States.
Trial summary
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye. In people with macular dystrophies, some of the cells in the macula gradually stop working and may die over time. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- What is being tested?
- ASP2020
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 4 sitesUnited States
- Study dates
- 14 Aug 2026to 30 Sep 2029
- Phase
- Phase 1
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Documented clinical diagnosis of macular dystrophy with a STGD-type clinical presentation and molecular confirmation, defined as either:
- STGD: presence of biallelic (pathogenic or likely pathogenic) ABCA4 variants, or one definite disease-causing ABCA4 variant together with a typical phenotype consistent with STGD.
- STGD-like macular dystrophy: presence of one or more pathogenic variants in a gene known to cause macular dystrophy, as appropriate for its expected inheritance mode.
- Sufficiently clear ocular media and adequate pupillary dilation to allow for all imaging procedures.
- Intraocular pressure (IOP) of ≤ 21 mmHg
- Participant has a spherical equivalent refractive error between +8.00 D and -10.00 D.
Show all 9Show fewer
- BCVA ranging from 20/500 to 20/40 (equivalent to 15 to 70 ETDRS letters)
- For participants in the > 20/80 to ≤ 20/40 BCVA range (moderate visual impairment [MVI]): presence of a visible definite or probable residual ellipsoid zone (EZ) on SD-OCT, and a total retinal SD-OCT central subfield thickness ≥ 150 micrometers (µm)
- For participants in the ≥ 20/500 to ≤ 20/80 BCVA range (severe visual impairment): Presence of a residual ONL within the macular optical coherence tomography (OCT) scan area and Evidence of RPE disease/damage by means of SD-OCT (hypertransmission defect) and/or FAF imaging (questionably decreased autofluorescence/definitely decreased autofluorescence).
Who cannot join
- Participant has a known history of significant systemic disease that could impact ocular health or confound study assessments, based on medical history or prior clinical documentation.
- Participant has an autoimmune condition that requires treatment with immunomodulatory therapy and/or biologics that cause immunosuppression.
- Participant has known diagnosis of diabetes mellitus with a documented glycated hemoglobin (HbA1c) value ≥ 7% 3 months prior to screening and based on available medical records.
- Participant has a history or evidence of severe cardiac disease, cardiovascular or cerebrovascular disease, including a history of stroke within 12 months prior to screening.
- Participant has any complicating systemic disease or active malignancy.
- Participant has a known history of any systemic or metabolic condition, or physical examination finding that may significantly affect ocular health or interfere with the interpretation of study assessments.
Show all 26Show fewer
- Participant has presence of another known or suspected molecular diagnosis of macular or retinal disease that could confound interpretation of study outcomes, indicate a second concomitant retinal condition, or suggest a different etiology for the macular disease.
- Participant has macular atrophy due to any cause other than a genetically or clinically confirmed diagnosis of STGD or STGD-like macular dystrophy.
- Participant has evidence or history of choroidal neovascularization.
- Participant has diagnosis of any form of uncontrolled glaucoma (for high-tension glaucoma IOP > 25 mmHg).
- Participant has a history of steroid-induced IOP elevation or known steroid responder status.
- Participant has and/or is receiving treatment for thyroid eye disease.
- Participant has diabetic retinopathy in excess of mild nonproliferative diabetic retinopathy
- Participant has any other disease(s) affecting the optic nerve.
- Participant has a history of anterior or posterior uveitis and/or presence of intraocular inflammation (trace anterior chamber cell or flare), or history of idiopathic or autoimmune-associated uveitis in either eye.
- Participant has media opacities impeding the visualization of the fundus and/or the reliable performance of the visual function tests required by the protocol.
- Participant has aphakia.
- Participant has a clinically significant epiretinal membrane or evidence of clinically significant vitreomacular traction syndrome.
- Participant has any other disorders which could interfere with or confound visual acuity and other ocular assessments, including OCT or FAF.
- Participant has history of any of the following procedures: posterior vitrectomy, retinal detachment surgery, glaucoma filtering surgery, glaucoma drainage device implantation, selective laser trabeculoplasty, full-thickness or partial- thickness corneal transplant.
- Participant has had any intraocular surgery within 3 months of screening.
- Participant has a history of intraocular metallic foreign bodies.
- Participant has received any treatment including gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or any prior intravitreal treatment for any indication in either eye that may be considered to potentially interfere with the study participation or its conduct.
- Participant has received within 1 month prior to screening or is receiving concomitant treatment with any ocular or systemic medication known to be toxic to the lens, retina, or optic nerve.
- Participant has received any investigational therapy within 3 months prior to screening.
- Participant has any condition, which makes the participant unsuitable for study participation.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Cincinnati Eye Institute
Cincinnati, Ohio, 45242
- Recruiting
Retina Foundation of Southwest
Dallas, Texas, 75231
- Recruiting
Associated Retina Consultants
Phoenix, Arizona, 85020
- Recruiting
Deep Blue Retina
Southaven, Mississippi, 38671
About this study
Official title: A Phase 1b, Open-label, Multicenter Dose-escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Subretinal Dose of ASP2020 in Participants With Macular Dystrophies With a Stargardt-type Clinical Presentation
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye. In people with macular dystrophies, some of the cells in the macula gradually stop working and may die over time. This leads to loss of central vision, which can make it harder to read, recognize faces or see fine details. What's seen out of the corner of the eye (peripheral vision) is mostly unaffected. Stargardt disease (STGD) is a type of macular dystrophy which is caused by 1 faulty gene (ABCA4). Vision loss most typically begins in childhood or teenage years but may also develop in adulthood. As well as STGD, there are other macular dystrophies that look very similar to STGD and are called STGD-like macular dystrophies. These are caused by many other different genes. Together, STGD and STGD-like conditions can be called STGD-type macular dystrophies.
This is an early development study of ASP2020 in adults, teenagers, and children with STGD-type macular dystrophies. ASP2020 are human stem cells which have been changed into cells found in the macula. In this study ASP2020 will be given to people for the first time. The main aim of the study is to check the safety of ASP2020 and how well people tolerate it. Other aims are to learn if people have an immune reaction to ASP2020, and if there are signs that the stem cells replace damaged cells in the retina, and vision improves for people with STGD-type macular dystrophies.
Read more
ASP2020 will be given as a single injection into the eye, under the retina. This requires a surgical procedure where the person is put to sleep by a general anesthetic. At the end of surgery, a steroid will be injected into the eye to reduce any swelling.
The study has 2 parts. In Part 1, different small groups will receive a lower to higher dose of ASP2020. This is done to find a suitable dose to use in Part 2. The adults will receive the lower dose and higher dose before the teenagers. There will be a 6-month gap between the last adult receiving the lower dose of ASP2020 and the first teenager receiving the same lower dose. This will also happen for the last adult receiving the higher dose of ASP2020 and the first teenager receiving the higher dose of ASP2020. Any medical problems will be recorded for each dose in each group. Children will not receive ASP2020 in Part 1.
In Part 2, different groups of adults, teenagers and children will receive the most suitable dose of ASP2020 worked out from Part 1.
People will be in the study for about 1 year and they will visit the clinic several times. In both parts of the study, safety checks will be done at each visit, and the study doctors will continue to check for any medical problems throughout the study. Various eye tests and eye imaging will be done throughout the study. Blood tests will also be done at some of the visits during the study.
- Being tested
- ASP2020
- Keywords
- Macular Dystrophies, Stargardt type, ABCA4 related STGD, STGD-like macular dystrophies, Safety, STGD, STGD-like, STGD-type
- Medical subject headings
- Stargardt Disease, Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASP2020Drug
Subretinal Injection
| Study group | Role | What they receive |
|---|---|---|
| Part 1 Dose Escalation: Adult - Low Dose | Experimental | Adult participants will receive a single low dose of ASP2020 on Day 1, administered by subretinal injection as part of a surgical procedure, followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. Receives: Drug: ASP2020 |
| Part 1 Dose Escalation: Adult - High Dose | Experimental | Adult participants will receive a single high dose of ASP2020 on Day 1, administered by subretinal injection as part of a surgical procedure, followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. Receives: Drug: ASP2020 |
| Part 1 Dose Escalation: Adolescent - Low Dose | Experimental | Adolescent participants will receive single low dose of ASP2020 by subretinal injection on Day 1 followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. Receives: Drug: ASP2020 |
| Part 1 Dose Escalation: Adolescent - High Dose | Experimental | Adolescent participants will receive single high dose of ASP2020 by subretinal injection on Day 1 followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. Receives: Drug: ASP2020 |
| Part 2 Dose Expansion: Adult with macular dystrophies | Experimental | Adult participants will receive single optimal dose of ASP2020 established in part 1 by subretinal injection on Day 1 followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. Receives: Drug: ASP2020 |
| Part 2 Dose Expansion: Pediatric with macular dystrophies | Experimental | Pediatric participants will receive single optimal dose of ASP2020 established in part 1 by subretinal injection on Day 1 followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. Receives: Drug: ASP2020 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent adverse events (TEAEs)
An adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
TEAEs are defined as an AE observed after administration of ASP2020 or pre-existing AE that worsen in severity or frequency following administration of ASP2020.
Measured: Up to 52 weeks
Number of participants with serious adverse events (SAEs)
An SAE is defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or other medically important events.
Measured: Up to 52 weeks
Number of participants with adverse events of special interest (AESIs)
AESIs include, but are not limited to, the following:
- Ectopic, excessive, or aberrant cell growth of ASP2020, including proliferative changes
- Immune-mediated reactions, including unexpected or clinically significant inflammatory responses
- Any new diagnosis of malignancy
- Any clinically significant AEs possibly or definitely related to ASP2020
- Clinically significant AEs related to the surgical administration procedure
Measured: Up to 52 weeks
Number of participants with greater than or equal to 15 letter loss in best corrected visual acuity (BCVA) from baseline
BCVA will be measured from the Early Treatment of Diabetic Retinopathy Study (ETDRS) letters chart.
Measured: Up to 52 weeks
Number of participants with significant changes in vital signs from baseline
Number of participants with significant changes in vital signs from baseline will be reported.
Measured: Up to 52 weeks
Number of participants with significant changes in laboratory values from baseline
Number of participants with significant changes in laboratory values from baseline will be reported.
Measured: Up to 52 weeks
Secondary outcomes (14)
Change from baseline in BCVA
BCVA will be measured from the ETDRS letters chart.
Measured: Baseline and week 26, 52 or Early Termination (ET) visit whichever occurs first
Change from baseline in low luminance visual acuity (LLVA)
LLVA will be measured from the ETDRS letters chart.
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in Minnesota Reading Acuity Chart (MNREAD) parameters
MNREAD evaluates near reading acuity, reading speed and critical print size.
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in mesopic macular sensitivity
Mean sensitivity, point-wise sensitivity (PWS), scotomatous/non-seen point count) will be measured by mesopic microperimetry.
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in central retinal thickness (CRT)
CRT will be measured by spectral- domain optical coherence tomography (SD-OCT).
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in total photoreceptor thickness (TPT)
TPT will be measured by SD-OCT.
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in ellipsoid zone (EZ) integrity
EZ integrity will be measured by SD- OCT.
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in outer nuclear layer (ONL)
ONL will be measured by SD-OCT.
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in retinal pigment epithelium (RPE) integrity
RPE structural integrity will be measured by SD-OCT.
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in questionably decreased autofluorescence (QDAF)
QDAF will be measured with fundus autofluorescence (FAF).
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in definitely decreased autofluorescence (DDAF)
DDAF will be measured with FAF.
Measured: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in anti-human leukocyte antigen (HLA) antibodies
Anti-HLA antibodies will be measured from the serum samples.
Measured: Baseline and week 4, 12 and 52 or ET visit whichever occurs first
Change from baseline in anti herpes simplex virus thymidine kinase (HSV- TK) antibodies
Anti HSV-TK antibodies will be measured from the serum samples.
Measured: Baseline and week 4, 12 and 52 or ET visit whichever occurs first
Change from baseline in cytokines
Cytokines will be measured from the serum samples.
Measured: Baseline and week 1, 4, 12 and 52 or ET visit whichever occurs first
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2026
- Study start14 Aug 2026
- Primary completion30 Sep 2029 (estimated)
- Study completion30 Sep 2029 (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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Astellas Institute for Regenerative Medicine
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Lead, Study director, Astellas Institute for Regenerative Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07734064
- Sponsor's protocol ID
- 2020-CL-0101
- Status last verified
- Oct 2026
- First submitted
- 15 Jul 2026
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Open-label, Multicenter Dose-escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Subretinal Dose of ASP2020 in Participants With Macular Dystrophies With a Stargardt-type Clinical Presentation
- ClinicalTrials.gov ID
- NCT07734064
- Acronym / sponsor ID
- 2020-CL-0101
- Keywords
- Macular Dystrophies, Stargardt type, ABCA4 related STGD, STGD-like macular dystrophies, Safety, STGD, STGD-like, STGD-type
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Astellas Institute for Regenerative MedicineIndustry (pharma, biotech, devices)
- Study start
- 14 Aug 2026 (actual)
- Primary completion
- 30 Sep 2029 (estimated)
- Study completion
- 30 Sep 2029 (estimated)
- First posted
- 29 Jul 2026 (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.
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye. In people with macular dystrophies, some of the cells in the macula gradually stop working and may die over time. This leads to loss of central vision, which can make it harder to read, recognize faces or see fine details. What's seen out of the corner of the eye (peripheral vision) is mostly unaffected. Stargardt disease (STGD) is a type of macular dystrophy which is caused by 1 faulty gene (ABCA4). Vision loss most typically begins in childhood or teenage years but may also develop in adulthood. As well as STGD, there are other macular dystrophies that look very similar to STGD and are called STGD-like macular dystrophies. These are caused by many other different genes. Together, STGD and STGD-like conditions can be called STGD-type macular dystrophies.
This is an early development study of ASP2020 in adults, teenagers, and children with STGD-type macular dystrophies. ASP2020 are human stem cells which have been changed into cells found in the macula. In this study ASP2020 will be given to people for the first time. The main aim of the study is to check the safety of ASP2020 and how well people tolerate it. Other aims are to learn if people have an immune reaction to ASP2020, and if there are signs that the stem cells replace damaged cells in the retina, and vision improves for people with STGD-type macular dystrophies.
ASP2020 will be given as a single injection into the eye, under the retina. This requires a surgical procedure where the person is put to sleep by a general anesthetic. At the end of surgery, a steroid will be injected into the eye to reduce any swelling.
The study has 2 parts. In Part 1, different small groups will receive a lower to higher dose of ASP2020. This is done to find a suitable dose to use in Part 2. The adults will receive the lower dose and higher dose before the teenagers. There will be a 6-month gap between the last adult receiving the lower dose of ASP2020 and the first teenager receiving the same lower dose. This will also happen for the last adult receiving the higher dose of ASP2020 and the first teenager receiving the higher dose of ASP2020. Any medical problems will be recorded for each dose in each group. Children will not receive ASP2020 in Part 1.
In Part 2, different groups of adults, teenagers and children will receive the most suitable dose of ASP2020 worked out from Part 1.
People will be in the study for about 1 year and they will visit the clinic several times. In both parts of the study, safety checks will be done at each visit, and the study doctors will continue to check for any medical problems throughout the study. Various eye tests and eye imaging will be done throughout the study. Blood tests will also be done at some of the visits during the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Part 1 Dose Escalation: Adult - Low Dose | Experimental | Drug: ASP2020Adult participants will receive a single low dose of ASP2020 on Day 1, administered by subretinal injection as part of a surgical procedure, followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. |
| Part 1 Dose Escalation: Adult - High Dose | Experimental | Drug: ASP2020Adult participants will receive a single high dose of ASP2020 on Day 1, administered by subretinal injection as part of a surgical procedure, followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. |
| Part 1 Dose Escalation: Adolescent - Low Dose | Experimental | Drug: ASP2020Adolescent participants will receive single low dose of ASP2020 by subretinal injection on Day 1 followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. |
| Part 1 Dose Escalation: Adolescent - High Dose | Experimental | Drug: ASP2020Adolescent participants will receive single high dose of ASP2020 by subretinal injection on Day 1 followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. |
| Part 2 Dose Expansion: Adult with macular dystrophies | Experimental | Drug: ASP2020Adult participants will receive single optimal dose of ASP2020 established in part 1 by subretinal injection on Day 1 followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. |
| Part 2 Dose Expansion: Pediatric with macular dystrophies | Experimental | Drug: ASP2020Pediatric participants will receive single optimal dose of ASP2020 established in part 1 by subretinal injection on Day 1 followed by triamcinolone acetonide as adjunct treatment by posterior sub-Tenon injection. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Documented clinical diagnosis of macular dystrophy with a STGD-type clinical presentation and molecular confirmation, defined as either:
- Sufficiently clear ocular media and adequate pupillary dilation to allow for all imaging procedures.
- Intraocular pressure (IOP) of ≤ 21 mmHg
- Participant has a spherical equivalent refractive error between +8.00 D and -10.00 D.
- BCVA ranging from 20/500 to 20/40 (equivalent to 15 to 70 ETDRS letters)
Key exclusion criteria (26 listed)
- Participant has a known history of significant systemic disease that could impact ocular health or confound study assessments, based on medical history or prior clinical documentation.
- Participant has an autoimmune condition that requires treatment with immunomodulatory therapy and/or biologics that cause immunosuppression.
- Participant has known diagnosis of diabetes mellitus with a documented glycated hemoglobin (HbA1c) value ≥ 7% 3 months prior to screening and based on available medical records.
- Participant has a history or evidence of severe cardiac disease, cardiovascular or cerebrovascular disease, including a history of stroke within 12 months prior to screening.
- Participant has any complicating systemic disease or active malignancy.
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 |
|---|---|---|
| ASP2020Subretinal Injection | Drug | Part 1 Dose Escalation: Adolescent - High Dose, Part 1 Dose Escalation: Adolescent - Low Dose, Part 1 Dose Escalation: Adult - High Dose, Part 1 Dose Escalation: Adult - Low Dose, Part 2 Dose Expansion: Adult with macular dystrophies, Part 2 Dose Expansion: Pediatric with macular dystrophies |
Primary outcome
Number of participants with treatment-emergent adverse events (TEAEs)
Time frame: Up to 52 weeks
An adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. TEAEs are defined as an AE observed after administration of ASP2020 or pre-existing AE that worsen in severity or frequency following administration of ASP2020.
Number of participants with serious adverse events (SAEs)
Time frame: Up to 52 weeks
An SAE is defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or other medically important events.
Number of participants with adverse events of special interest (AESIs)
Time frame: Up to 52 weeks
AESIs include, but are not limited to, the following: * Ectopic, excessive, or aberrant cell growth of ASP2020, including proliferative changes * Immune-mediated reactions, including unexpected or clinically significant inflammatory responses * Any new diagnosis of malignancy * Any clinically significant AEs possibly or definitely related to ASP2020 * Clinically significant AEs related to the surgical administration procedure
Number of participants with greater than or equal to 15 letter loss in best corrected visual acuity (BCVA) from baseline
Time frame: Up to 52 weeks
BCVA will be measured from the Early Treatment of Diabetic Retinopathy Study (ETDRS) letters chart.
Number of participants with significant changes in vital signs from baseline
Time frame: Up to 52 weeks
Number of participants with significant changes in vital signs from baseline will be reported.
Number of participants with significant changes in laboratory values from baseline
Time frame: Up to 52 weeks
Number of participants with significant changes in laboratory values from baseline will be reported.
14 secondary outcomes
Change from baseline in BCVA
Time frame: Baseline and week 26, 52 or Early Termination (ET) visit whichever occurs first
Change from baseline in low luminance visual acuity (LLVA)
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in Minnesota Reading Acuity Chart (MNREAD) parameters
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in mesopic macular sensitivity
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in central retinal thickness (CRT)
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in total photoreceptor thickness (TPT)
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in ellipsoid zone (EZ) integrity
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in outer nuclear layer (ONL)
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in retinal pigment epithelium (RPE) integrity
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in questionably decreased autofluorescence (QDAF)
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in definitely decreased autofluorescence (DDAF)
Time frame: Baseline and week 26, 52 or ET visit whichever occurs first
Change from baseline in anti-human leukocyte antigen (HLA) antibodies
Time frame: Baseline and week 4, 12 and 52 or ET visit whichever occurs first
Change from baseline in anti herpes simplex virus thymidine kinase (HSV- TK) antibodies
Time frame: Baseline and week 4, 12 and 52 or ET visit whichever occurs first
Change from baseline in cytokines
Time frame: Baseline and week 1, 4, 12 and 52 or ET visit whichever occurs first
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 23 Oct 2026.
- Individual participant dataSharing planned: No. Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.
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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.
Medical Lead study director
Astellas Institute for Regenerative Medicine
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ASP2020 in people with Stargardt Disease and Stargardt Macular Dystrophy, run by Astellas Institute for Regenerative Medicine at 4 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 9 inclusion and 26 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 23 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 4 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 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.
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 Astellas Institute for Regenerative Medicine (800-888-7704, Astellas.registration@astellas.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-emergent adverse events (TEAEs) (time frame: Up to 52 weeks). The study lists 6 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 23 Oct 2026.