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Actively Recruiting
Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease
About
Brief Summary
The purpose of this study is to determine the long term safety and tolerability of ALK-001 (C20-D3-retinyl acetate), and to explore the effects of ALK-001 on the progression of Stargardt disease in patients between the ages of 8 and 70 years old.
Funding Source - FDA OOPD
Primary Purpose
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
Simplified Inclusion Criteria:
- Male or female between 8 and 70 years old (inclusive), with any visual acuity
- Has a clinical diagnosis of typical autosomal recessive Stargardt macular dystrophy (STGD1)
- Has provided a genetic report indicating at least two ABCA4 disease-causing mutations. When only one ABCA4 disease-causing mutation is reported, sponsor's permission will be required.
- At least one eye (called the "primary study eye") must have at least one well-demarcated area of significantly reduced autofluorescence as imaged by fundus autofluorescence (FAF), have decreased retinal sensitivity as measured by microperimetry, or have maculopathy expected to progress over the duration of the study
- Primary study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging
- Healthy as judged by investigator
- Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study
- Has signed and dated the informed consent forms (or assent where appropriate) to participate
- Female of childbearing potential has signed the informed consent about birth defects or attestation on contraception requirements
Main Exclusion Criteria:
- Has taken disallowed items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days
- Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization
- Has concurrent medical condition or history, which in the opinion of the investigator, is likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or study procedures
- Has clinically significant abnormal laboratory result(s) at screening
- Has active or historical acute or chronic liver disorder
- Has active or historical ocular disorder in the primary study eye that, in the opinion of the investigator, may confound assessment of the retina morphologically or functionally (this could include for example cataract surgery within the past 6 months, choroidal neovascularization (CNV), glaucoma, recurring uveitis, diabetic retinopathy, other retinal disease, etc.)
- Has had intraocular surgery or injections in the primary study eye within 90 days of the screening visit
- Has a clinically significant abnormal electrocardiogram (ECG), or has a corrected QT interval (QTc) that is 450 ms or greater
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Study Stats
Protocol No.
16-000116
Category
Brain/Neurological Diseases
Eye/Ocular Disorders
Principal Investigator
Contact
Location
- UCLA Westwood