Actively Recruiting

Age: 6Years +
All Genders
ID07425574

Observational Study of Disease Progression in Participants With Stargardt-type Macular Dystrophies

Led by Astellas Pharma Global Development, Inc. · Updated on 2026-05-28

90

Participants Needed

7

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are studying Stargardt disease (STGD) and similar inherited eye conditions known as STGD-type macular dystrophies, which affect the macula, the central part of the retina responsible for sharp vision. These conditions cause central vision loss over time while mostly sparing side vision. The study aims to understand if the disease progresses similarly in people with STGD and those with related macular dystrophies caused by different genes. Genetic testing is the only way to confirm the specific condition. Participants with STGD or STGD-like macular dystrophies will continue their usual care with their own doctors. They will visit the clinic every 6 months for up to 2 years to undergo standard eye tests and imaging. Medical records and previous clinical data from the last 24 months will also be reviewed when available. No investigational treatments are given, as this is an observational study. During the study, participants will have their vision and eye structure assessed using tests like best corrected visual acuity (BCVA) and imaging. Researchers will collect information on participants' wellbeing, general health, medications, supplements, and daily activities. The main outcome measured is the change in BCVA over 12 months, with additional assessments at months 6, 18, and 24. Safety and disease progression will be monitored throughout the study period.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 6Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participant has a documented clinical diagnosis of macular dystrophy with a Stargardt-type presentation confirmed by genetic testing
  • Participant has clear eye media and pupils dilate adequately for imaging
  • Intraocular pressure is 21 mmHg or less at screening and baseline
  • Spherical equivalent refractive error between +8.00 D and -10.00 D, with exceptions for high myopia if no degenerative changes
  • Best corrected visual acuity (BCVA) between 20/500 and 20/40
  • Evidence of retinal pigment epithelium disease/damage on imaging
  • Evidence of decreased autofluorescence on retinal imaging
  • Ability and willingness to comply with study visits and assessments
  • Capability to perform all required eye tests reliably
Not Eligible

You will not qualify if you...

  • History of significant systemic diseases that affect eye health or study assessments
  • Autoimmune conditions requiring immunosuppressive therapy
  • Diabetes with hemoglobin A1c of 7% or higher unless no diabetic complications
  • Severe cardiac, cardiovascular, or cerebrovascular diseases within recent months
  • Presence of other retinal or macular diseases that could affect study results
  • Uncontrolled glaucoma or thyroid eye disease
  • Diabetic retinopathy beyond mild stage
  • History of uveitis or eye inflammation
  • Corneal or lens opacities affecting imaging or eye tests
  • Eye anatomy issues preventing pupil dilation
  • Other ocular disorders interfering with vision or imaging
  • Prior eye surgeries or treatments that could affect study participation
  • Use of medications toxic to the eye or recent experimental treatments
  • Recent major surgeries or planned surgeries during the study
  • Known hypersensitivity to eye drops used in testing
  • Any condition making the participant unsuitable for the study

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Long-term Monitoring

Duration - Up to 24 months

Participants with Stargardt-type eye conditions are observed over time to monitor disease progression through regular eye assessments.

Visits at baseline, and Months 6, 12, 18, and 24 with additional visits for early termination if needed

Trial Site Locations

Total: 7 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

University of California Health - UC Davis

Sacramento, California, United States, 95817

Actively Recruiting

4

Deep Blue Retina

Southaven, Mississippi, United States, 38671

Actively Recruiting

5

Duke Eye Center

Durham, North Carolina, United States, 27710

Actively Recruiting

6

Casey Eye Institute - Oregon Health & Science University

Portland, Oregon, United States, 97239

Actively Recruiting

7

Retina Foundation of the Southwest

Dallas, Texas, United States, 75231

Actively Recruiting

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Research Team

A

Astellas Pharma Global Development, Inc.

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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