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Stargardt DiseaseEVOLVENCT07425574

A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults

An observational study of Stargardt Disease and Stargardt Macular Dystrophy, run by Astellas Pharma Global Development, Inc. at 15 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

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
How many people
90 participants
Where
15 sitesUnited States
Study dates
20 Feb 2026to 31 Dec 2028
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 6 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: Participants aged ≥ 6 years at baseline with clinically confirmed macular dystrophy of STGD-type clinical presentation

Who may be able to join

  • Participant has a documented clinical diagnosis of macular dystrophy expressing a STGD-type clinical presentation and molecular confirmation, defined as either:
  • ABCA4-associated disease: 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. Note: All genetic testing should be performed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory or equivalent whenever possible. Acceptable documentation includes a copy of the genetic test report, laboratory certification statement, or clinical notes explicitly referencing CLIA certification. Predicted pathogenic variant of uncertain significance (VUS) that cannot be confirmed by standard laboratory criteria as certainly disease-causing (novel mutations) will be considered on a case by-case basis.
  • Participant has sufficiently clear ocular media and adequate pupillary dilation to allow for all imaging procedures.
  • Participant has intraocular pressure (IOP) both at screening and baseline of ≤ 21 mmHg measured by applanation tonometry. Note: Participant who is on topical IOP lowering treatment may also be included.
  • Participant has a spherical equivalent refractive error between + 8.00 D and - 10.00 D. Eyes with high myopia (> -10.00 D) are permitted only if there are no signs of myopic degenerative macular changes.
Show all 15Show fewer
  • Participant has a BCVA ranging from 20/500 to 20/40 (equivalent to 15 to 70 ETDRS letters read at 1 meter)
  • For participants in the > 20/80 to ≤ 20/40 BCVA range (moderate visual impairment [MVI]): presence of a visible definite or probable residual EZ on spectral-domain optical coherence tomography (SD-OCT) (no minimum residue size required) and a total retinal SD-OCT central subfield retinal thickness ≥ 150 µm
  • For participants in the ≥ 20/500 to ≤ 20/80 BCVA range (SVI): presence of a residual outer nuclear layer (ONL) within the macular SD-OCT scan area and measurable within the Age-Related Eye Disease Study (AREDS) grading grid and no residual EZ or minimal thickness of the ONL required
  • Participant has evidence of retinal pigment epithelium (RPE) disease/damage on SD-OCT (hypertransmission defects [HTDs]) and/or FAF imaging.
  • Participant has evidence of at least questionably decreased autofluorescence (QDAF) (incomplete retinal pigment epithelial and outer retinal atrophy [iRORA]), defined as mottled or speckled hypo-autofluorescence (AF) signal, mixed with or without hyper- AF, on FAF imaging. Note: At study onset, having patches of definitely decreased autofluorescence (DDAF) within the FAF image and/or patches of complete retinal pigment epithelial and outer retinal atrophy (cRORA) on SD-OCT ≥ 250 µm will not be a requirement for participation but will be acceptable for inclusion, so long as:
  • DDAF/cRORA areas are contained within the limits of the AREDS grading grid (≤ 12 disc areas), and physically distinct and separate from other areas of QDAF, DDAF and/or hyper-AF that may be present outside the AREDS grading grid, and
  • the other BCVA and SD-OCT required inclusion criteria are also met.
  • Participant is able to comply with the protocol and willing to undertake all scheduled visits and assessments during the total study period.
  • Participant is deemed capable of performing reliably all tests required for participation.

Who cannot join

  • Participant has a known history of significant systemic disease (e.g., uncontrolled hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or immunocompromising conditions such as human immunodeficiency virus [HIV]) 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 a known diagnosis of diabetes mellitus with a documented hemoglobin A1c (HbA1c) value ≥ 7% 3 months prior to screening and based on available medical records. If the documented HbA1c is ≥ 7% and there is no clinical history of diabetic symptoms, diabetic retinopathy, abnormal renal function (e.g., elevated creatinine), or glycosuria noted in medical records, the participant may be enrolled.
  • 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.
  • Participant has a history or evidence of severe cardiac disease (e.g., New York Heart Association Functional Class III or IV), clinical evidence of unstable angina, acute coronary syndrome, myocardial infarction or revascularization within 6 months prior to screening.
  • Participant has a history or evidence of ventricular tachyarrhythmia requiring ongoing treatment.
Show all 39Show fewer
  • Participant has a significant cardiovascular or cerebrovascular disease, including a history of stroke within 12 months prior to screening.
  • Participant has any complicating systemic disease or active malignancy. Squamous cell carcinomas will be an exclusionary criterion if lesions are recurrent and/or > 2 cm in diameter and/or affect the lip or ear area. Basal cell carcinomas are permitted, provided they are not located in the immediate periocular facial region. A history of any prior malignancy is allowed if the treating physician or oncologist confirms that the participant has been free of recurrence or metastasis for at least 5 years prior to screening.
  • Participant has another known or suspected molecular diagnosis of macular or retinal disease (e.g., pathogenic mutations in other genes) that could confound interpretation of study outcomes, indicate a second concomitant retinal condition or suggest a different etiology for the macular disease.
  • Participant has evidence or history of choroidal neovascularization.
  • Participant has macular atrophy due to any cause other than a genetically or clinically confirmed diagnosis of STGD or STGD-like macular dystrophies.
  • Participant has a known diagnosis of any form of uncontrolled glaucoma (for high tension glaucoma IOP > 25 mmHg).
  • Participant has and/or is receiving treatment for thyroid eye disease.
  • Participant has diabetic retinopathy in excess of mild nonproliferative diabetic retinopathy (i.e., presents with widespread retinal microaneurysms, dot-blot hemorrhages, and cotton-wool retinal spots).
  • 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. Note: A single, sporadic episode of anterior uveitis without recurrences in the past 5 years and not associated with chronic conditions that increase the likelihood of recurrence may be accepted on a case-by-case basis in consultation with the sponsor's medical monitor/lead.
  • Participant has corneal or lenticular opacities impeding the visualization of the fundus and/or the reliable performance of the visual function tests required by the protocol.
  • Participant has sufficiently narrow iridocorneal angles contraindicating pupillary dilation that have not been previously treated with preventative laser iridotomies.
  • Participant has any other ocular disorders that could interfere with or confound visual acuity and other ocular assessments, including OCT or FAF.
  • Participant has a clinically significant epiretinal membrane or evidence of clinically significant vitreomacular traction syndrome. Note: A mild enhancement of the vitreoretinal interface seen on OCT without any alteration of the foveal profile would not be considered an exclusion criterion.
  • Participant has presence of or recurrent vitreous hemorrhages. Note: A one-time episode of a traumatic vitreous hemorrhage would not result in exclusion, so long as resolved.
  • Participant has aphakia.
  • Participant has anticipated need for cataract surgery during the study period.
  • Participant has a macular hole, evident by ophthalmoscopy and/or by SD-OCT examinations, or history of prior macular hole surgery.
  • Participant has a history of any of the following procedures: posterior vitrectomy, retinal detachment surgery, glaucoma filtering surgery (e.g., trabeculectomy), glaucoma drainage device implantation, selective laser trabeculoplasty, full-thickness or partial-thickness corneal transplant (e.g., Descemet's stripping endothelial keratoplasty, Descemet's stripping automated endothelial keratoplasty or Descemet's membrane endothelial keratoplasty).
  • Participant has any lesion that is considered to pose a risk of retinal detachment during the study. Any preexisting lesion may be treated to allow eligibility, but treatment must be completed at least 3 months prior to screening.
  • Participant has had any prior thermal laser or photodynamic therapy treatment in the macular region, regardless of indication. Peripheral retinal thermal laser treatments for holes, lattice degeneration, and/or other lesions with rhegmatogenous potential are permitted within 3 months prior to screening, subject to their potential impact on a future pars plana vitrectomy surgery.
  • Participant has had any intraocular surgery within 3 months of screening.
  • Participant has a history of intraocular metallic foreign bodies.
  • Participant has received (or plans to receive during the study) 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 is receiving concomitant treatment with any ocular or systemic medication known to be toxic to the lens, retina or optic nerve.
  • Participant is using hydroxychloroquine (Plaquenil), chloroquine, pentosan polyphosphate (Elmiron), tamoxifen, chlorpromazine, thioridazine (Mellaril), isotretinoin (Accutane) or any related retina-toxic compounds at screening.
  • Participant has received (or plans to receive during the study) therapeutic radiation in the region of the study eye.
  • Participant has had (or plans to have during the study) any major surgical procedure within 1 month prior to screening or anticipates having one during the study, which may interrupt study participation.
  • Participant is taking glucagon-like peptide-1 (GLP-1) receptor agonists and is unable or unwilling to stop the medication at least 4 weeks prior to screening. A history of treatment with a GLP-1 receptor agonist is allowed.
  • Participant is taking metformin and is unable or unwilling to stop this medication at least 4 weeks prior to screening. A history of treatment with metformin is allowed.
  • Participant is participating (or plans to participate) in an interventional study of a vitamin A metabolism inhibitor, a vitamin A analog/derivative, or another investigational drug product within 3 months prior to screening. After a 3 month washout period, participants can be considered for enrollment. Participation in a noninterventional study (NIS) is allowed.
  • Participant has a known or suspected hypersensitivity to eyedrops used for IOP measurement or pupil dilation.
  • Participant has any condition that makes the participant unsuitable for study participation.

Trial locations and contacts

15 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Astellas Pharma Global Development, Inc. contact

800-888-7704

Astellas.registration@astellas.com

15 sites in 1 country

United States15 sites, 15 recruiting

  • University of Michigan Health - Kellogg Eye Center

    Ann Arbor, Michigan, 48105

    Recruiting

  • University of Illinois Chicago - UI Health

    Chicago, Illinois, 60612

    Recruiting

  • Cincinnati Eye Institute

    Cincinnati, Ohio, 45242

    Recruiting

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231

    Recruiting

  • Duke Eye Center

    Durham, North Carolina, 27710

    Recruiting

  • University of Florida Health - UF Health Jacksonville

    Jacksonville, Florida, 32209

    Recruiting

  • University of Miami Health System - Bascom Palmer Eye Institute

    Miami, Florida, 33136

    Recruiting

  • Stanford University School of Medicine

    Palo Alto, California, 94303

    Recruiting

  • Wills Eye Physicians - Mid Atlantic Retina

    Philadelphia, Pennsylvania, 19107

    Recruiting

  • Associated Retina Consultants

    Phoenix, Arizona, 85020

    Recruiting

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213

    Recruiting

  • Casey Eye Institute - Oregon Health & Science University

    Portland, Oregon, 97239

    Recruiting

  • University of California Health - UC Davis

    Sacramento, California, 95817

    Recruiting

  • University of Utah Health - John A Moran Eye Center

    Salt Lake City, Utah, 84312

    Recruiting

  • Deep Blue Retina

    Southaven, Mississippi, 38671

    Recruiting

Other trials in United States

About this study

Official title: An Observational Study of Natural Disease Progression in Participants With Macular Dystrophies Expressing 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 vision loss in the center of the eye. Side vision (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 happens in childhood, but many people do not develop it until they are adults. As well as STGD, there are other macular dystrophies that look very similar to STGD but that are caused by many other different genes. Together, STGD and STGD-like conditions can be called STGD-type macular dystrophies. This is because they look the same clinically and have similar symptoms. Since different genes can cause these conditions, genetic testing is the only way to be sure which specific condition a person has.

In this study, researchers want to learn if the disease progresses in a similar way in people with STGD and STGD-like macular dystrophies. People taking part in the study will continue to manage their condition, as agreed with their own doctor. People will visit their clinic every 6 months to have various standard eye tests and imaging. The information collected will include questions about people's wellbeing, general health, medication and supplements taken, and daily activities.

Children over 6 years old and adults with STGD-type macular dystrophies may take part in this study. They will be in the study for up to 24 months (2 years). The study sponsor (Astellas) will not decide how people's condition is managed. However, the sponsor will provide instructions on when people visit their clinic and what is recorded during the study. If available, medical records, clinical and imaging data from previous visits going back 24 months will also be reviewed.

Being tested
No Intervention
Keywords
Macular Dystrophies, Stargardt type, ABCA4 related STGD, STGD-like macular dystrophies
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.

  • No InterventionOther

    No investigational drug will be administered to participants in this study.

Study groups in NCT07425574
Study groupRoleWhat they receive
Participants with Stargardt-type eye conditionsParticipants with STGD or STGD-like macular dystrophies.
Receives: Other: No Intervention

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from baseline in best corrected visual acuity (BCVA) at month 12

    BCVA will be measured by Early Treatment Diabetic Retinopathy Study (ETDRS) letters chart.

    Measured: Baseline and Month 12

Secondary outcomes (27)

  1. Change from baseline in BCVA

    BCVA will be measured by ETDRS letters chart.

    Measured: Baseline and Months 6, 18 and 24/Early Termination (ET)

  2. Change from baseline in low luminance visual acuity (LLVA)

    LLVA will be measured by ETDRS letters chart.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  3. Change from baseline in Minnesota Reading Acuity Chart (MNREAD)

    MNREAD evaluates reading acuity, critical print size and maximum reading speed.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  4. Change from baseline in mesopic MP sensitivity

    Defect-mapping, fundus-controlled microperimetry (MP) will be used to assess change in mesopic MP sensitivity.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  5. Difference between eyes in change from baseline in BCVA

    BCVA will be measured by ETDRS letters chart.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  6. Difference between eyes in change from baseline in LLVA

    LLVA will be measured by ETDRS letters chart.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  7. Change from baseline in central retinal thickness (CRT)

    CRT will be measured by spectral-domain optical coherence tomography (SD-OCT).

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  8. Change from baseline in total photoreceptor thickness (TPT)

    TPT will be measured by SD-OCT.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  9. Change from baseline in ellipsoid zone (EZ) integrity

    EZ integrity will be measured by SD-OCT.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  10. Change from baseline in QDAF (Questionably Diminished Auto-Fluorescence)

    QDAF will be measured with Fundus Auto-Fluorescence (FAF).

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  11. Change from baseline in DDAF (Definitely Diminished Auto-Fluorescence)

    DDAF will be measured with FAF.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  12. Difference between eyes in change from baseline in EZ integrity

    EZ integrity will be measured by SD-OCT.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  13. Difference between eyes in change from baseline in CRT

    CRT will be measured by SD-OCT.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  14. Difference between eyes in change from baseline in TPT

    TPT will be measured by SD-OCT.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  15. Difference between eyes in change from baseline in QDAF

    QDAF will be measured by FAF.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  16. Difference between eyes in change from baseline in DDAF

    DDAF will be measured by FAF.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  17. Correlation between change from baseline in structural outcomes and functional outcomes

    Structural outcomes include: CRT, TPT, EZ integrity and FAF. Functional outcomes include: BCVA, LLVA, MNREAD parameters and mesopic MP sensitivity.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  18. Longitudinal Association Between the Rate of Change in Structural and Functional Retinal Outcomes Assessed by Joint Modelling Analysis

    Change from baseline in structural outcomes (CRT, TPT, EZ Integrity) and functional outcomes (BCVA, Mesopic MP sensitivity) will be used for joint modelling analysis.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  19. Rate of Disease Progression Over Time Associated with Structural and Functional Characteristics at Baseline

    Baseline characteristics: Genotype (ABCA4 vs non-ABCA4), BCVA, presence and size of central EZ, CRT and TPT, mesopic MP sensitivity.

    Rate of disease progression over time: Change from baseline in BCVA, EZ area, CRT, TPT, FAF and MP sensitivity.

    Measured: Baseline and Months 6, 12, 18 and 24/ET

  20. Intraclass correlation coefficient at baseline (2 repeated measurements) in BCVA

    BCVA will be measured by ETDRS letters chart.

    Measured: Baseline

  21. Intraclass correlation coefficient longitudinally between baseline and Month 12 in BCVA

    BCVA will be measured by ETDRS letters chart.

    Measured: Baseline and Month 12

  22. Intraclass correlation coefficient at baseline (2 repeated measurements) in LLVA

    LLVA will be measured by ETDRS letters chart.

    Measured: Baseline

  23. Intraclass correlation coefficient longitudinally between baseline and Month 12 in LLVA

    LLVA will be measured by ETDRS letters chart.

    Measured: Baseline and Month 12

  24. Intraclass correlation coefficient at baseline (2 repeated measurements) in MNREAD parameters

    MNREAD evaluates reading acuity, critical print size and maximum reading speed.

    Measured: Baseline

  25. Intraclass correlation coefficient longitudinally between baseline and Month 12 in MNREAD parameters

    MNREAD evaluates reading acuity, critical print size and maximum reading speed.

    Measured: Baseline and Month 12

  26. Intraclass correlation coefficient at baseline (2 repeated measurements) in mesopic MP sensitivity

    MP will be used to assess mesopic sensitivity.

    Measured: Baseline

  27. Intraclass correlation coefficient longitudinally between baseline and Month 12 in mesopic MP sensitivity

    MP will be used to assess mesopic sensitivity.

    Measured: Baseline and Month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Feb 2026
  2. Study start20 Feb 2026
  3. Primary completion31 Dec 2028 (estimated)
  4. Study completion31 Dec 2028 (estimated)

Resources

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
Prospective
Enrolment
90 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07425574
Sponsor's protocol ID
2020-CL-0102
Acronym
EVOLVE
Status last verified
Oct 2026
First submitted
2 Feb 2026
Last updated
20 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
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/.

Questions about this trial

What is this clinical trial studying?

An observational study of Stargardt Disease and Stargardt Macular Dystrophy, run by Astellas Pharma Global Development, Inc. at 15 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 15 inclusion and 39 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 20 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 15 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 Astellas Pharma Global Development, Inc. (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?

Change from baseline in best corrected visual acuity (BCVA) at month 12 (time frame: Baseline and Month 12). 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 NCT07425574.

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 20 Oct 2026.

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