Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
Condition: Stargardt Disease; Macular Atrophy
MeSH terms: Stargardt Disease; Macular Degeneration; Anetoderma; Atrophy
Intervention: Emixustat
Intervention description: Once daily, tablet for oral administration
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 23.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy
      secondary to Stargardt disease.
    

Eligibility criteria:

        Inclusion Criteria, including, but not limited to:

          -  Clinical diagnosis of macular atrophy (MA) secondary to Stargardt disease (STGD) in
             one or both eyes

          -  At least 2 pathogenic mutations of the ABCA4 gene

          -  Early Treatment Diabetic Retinopathy Study BCVA of ≥ 20 letters (approximately ≥
             20/400 Snellen) in the study eye

          -  Adequate clarity of ocular media and adequate pupillary dilation to permit good
             quality imaging of MA in the study eye

          -  Able and willing to provide written informed consent before undergoing any
             study-related procedures

          -  Able to reliably administer oral medication by self or with available assistance

        Exclusion Criteria, including, but not limited to:

          -  Macular atrophy associated with a condition other than STGD in either eye.

          -  Presence in either eye of an active ocular disease that in the opinion of the
             Investigator compromises or confounds visual function.

          -  History of any intraocular or ocular surface surgery in either eye within 3 months of
             screening.

          -  Current or previous participation in an interventional study to treat STGD using gene
             therapy or stem cell therapy at any time, or participation in an interventional study
             of a vitamin A derivative ≤3 months prior to screening.

          -  Pre-specified laboratory abnormalities at screening

          -  Presence of other medical or ophthalmic disease, physical examination finding, or
             clinical laboratory finding that in the opinion of the Investigator may contraindicate
             the use of an investigational drug and place the subject at risk

          -  Current or history of cancer (except for adequately treated basal cell or squamous
             cell carcinoma of the skin) within 1 year of screening

          -  History of myocardial infarction, stroke, unstable ischemic heart disease,
             uncontrolled cardiac arrhythmia, or hospitalization for congestive heart failure
             within 6 months of screening.

          -  Anticipated hospitalization for a medical/surgical procedure(s) that could result in
             interruption/premature cessation of study treatment or participation.

          -  Electrocardiogram with a clinically significant abnormal finding

          -  Female subjects who are pregnant or lactating

          -  Female subjects of childbearing potential or male subjects who are not surgically
             sterile who are not willing to practice a medically accepted method of birth control
             with their sexual partner from screening through 30 days after the final dose of study
             drug