Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Foundation Fighting Blindness
With
United States Department of Defense
Age
6–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Provide a signed informed consent form and authorization allowing the disclosure and use of protected health information.
The designated primary study eye must have at least one well-demarcated area of atrophy as imaged by fundus autofluorescence with a minimum diameter of 300 microns and all lesions together must add to less than or equal to 12 mm2 (equivalent to no more than 5 disc areas in a least one eye) and a BCVA of 20 ETDRS letters (20/400 Snellen equivalent) or better.
Two (2) pathogenic mutations confirmed present, in the ABCA4 gene. If only one ABCA4 allele contains a pathogenic mutation, the patient shall have a typical Stargardt phenotype, namely at least one eye must have flecks at the level of the retinal pigment epithelium typical for STGD.
The primary study eye must have clear ocular media and adequate pupillary dilation to permit good quality fundus autofluorescence (FAF) and Spectral-Domain optical coherence tomography (sd-OCT) imaging in the opinion of the investigator.
Be able to cooperate in performing the examinations.
Be willing to undergo ocular examinations once every 6 months for up to 24 months.
Be at least six years old.
You may not be if
Ocular disease, such as choroidal neovascularization, glaucoma and diabetic retinopathy, in either eye that may confound assessment of the retina morphologically and functionally.
Intraocular surgery in the primary study eye within 90 days prior to baseline visit.
Current or previous participation in an interventional study to treat STGD such as gene therapy or stem cell therapy. Current participation in a drug trial or previous participation in a drug trial within six months before enrollment. The use of oral supplements of vitamins and minerals are permitted although the current use of Vitamin A supplementation shall be documented.
The site Principal Investigator may declare any patient at their site ineligible to participate in the study for a sound medical reason prior to the patient's enrollment into the study.
Any systemic disease with a limited survival prognosis (e.g. cancer, severe/unstable cardiovascular disease).
Any condition that would interfere with the patient attending their regular follow-up visits every 6 months for up to 24 months, e.g. personality disorder, use of major tranquilizers such as Haldol or Phenothiazine, chronic alcoholism, Alzheimer's Disease or drug abuse.
Evidence of significant uncontrolled concomitant diseases such as cardiovascular, neurological, pulmonary, renal, hepatic, endocrine or gastro-intestinal disorders.
Clareo Health | A Natural History of the Progression of Stargardt Disease: Retrospective and Prospective Studies
