Actively Recruiting
A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults
Led by Astellas Pharma Global Development, Inc. · Updated on 2026-05-07
90
Participants Needed
4
Research Sites
127 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant has a documented clinical diagnosis of macular dystrophy with Stargardt-type presentation confirmed by molecular testing
- Presence of biallelic or one definite disease-causing ABCA4 variant with typical Stargardt phenotype or other pathogenic variants causing macular dystrophy
- Clear ocular media and adequate pupil dilation for imaging
- Intraocular pressure at screening and baseline of 21 mmHg or less, including those on topical eye pressure treatment
- Spherical equivalent refractive error between +8.00 D and -10.00 D, with allowance for high myopia if no degenerative macular changes
- Best corrected visual acuity (BCVA) between 20/500 and 20/40
- For moderate visual impairment (BCVA >20/80 to ≤20/40): visible residual ellipsoid zone on OCT and minimum central retinal thickness ≥150 µm
- For severe visual impairment (BCVA ≥20/500 to ≤20/80): residual outer nuclear layer on OCT within the macular area
- Evidence of retinal pigment epithelium disease on OCT or fundus autofluorescence imaging
- Evidence of questionably decreased autofluorescence with specific imaging characteristics
- Ability to comply with study protocol and attend all scheduled visits
- Capability to reliably perform required tests
You will not qualify if you...
- History of significant systemic diseases affecting eye health or study assessments, including uncontrolled hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease, or immunocompromising conditions like HIV
- Autoimmune conditions requiring immunosuppressive treatments
- Diabetes with HbA1c ≥7% unless no diabetic symptoms or complications
- Systemic or metabolic conditions affecting ocular health or study interpretation
- Severe cardiac disease or recent cardiac events within 6 months
- Ventricular tachyarrhythmia requiring ongoing treatment
- Significant cardiovascular or cerebrovascular disease, including stroke within 12 months
- Active malignancy or complicating systemic disease, with exceptions for certain cancers if disease-free for 5 years
- Other molecularly diagnosed macular or retinal diseases that could confound study results
- Evidence or history of choroidal neovascularization
- Macular atrophy from causes other than confirmed Stargardt-type dystrophies
- Uncontrolled glaucoma or eye diseases affecting optic nerve
- Thyroid eye disease or diabetic retinopathy beyond mild stage
- History or presence of uveitis or intraocular inflammation
- Corneal or lenticular opacities interfering with eye examinations
- Narrow iridocorneal angles contraindicating pupil dilation
- Other ocular disorders interfering with vision or imaging assessments
- Significant epiretinal membrane or vitreomacular traction syndrome
- Recurrent vitreous hemorrhages or aphakia
- Need for cataract surgery during study
- Macular hole or history of macular hole surgery
- History of major ocular surgeries including vitrectomy, retinal detachment, glaucoma surgeries, or corneal transplants
- Lesions posing retinal detachment risk unless treated at least 3 months prior
- Prior laser or photodynamic therapy in macular region
- Intraocular surgery within 3 months
- History of intraocular metallic foreign bodies
- Prior gene therapy, stem cell therapy, retinal chip implants, or intravitreal treatments affecting study
- Use of medications toxic to eye structures
- Therapeutic radiation near study eye
- Major surgery within 1 month or planned that could interrupt participation
- Use of GLP-1 receptor agonists or metformin unwilling to stop 4 weeks before screening
- Participation in investigational drug studies within 3 months without washout
- Hypersensitivity to eyedrops used for eye pressure or dilation
- Any other condition making participation unsuitable
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Associated Retina Consultants
Phoenix, Arizona, United States, 85020
Actively Recruiting
2
Stanford University School of Medicine
Palo Alto, California, United States, 94303
Actively Recruiting
3
Duke Eye Center
Durham, North Carolina, United States, 27710
Actively Recruiting
4
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
Research Team
A
Astellas Pharma Global Development, Inc.
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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