The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease. This study is an extension of NCT02402660 and enrolls participants who are at least 8 years old. Enrollment is by invitation only. Funding Source - FDA OOPD
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
200
Oral administration of a pill for up to 24 months
Alkeus Site
Phoenix, Arizona, United States
Alkeus Site
Los Angeles, California, United States
Alkeus Site
Aurora, Colorado, United States
Alkeus Site
Gainesville, Florida, United States
Alkeus Site
Indianapolis, Indiana, United States
Alkeus Site
Grand Rapids, Michigan, United States
Alkeus Site
Westbury, New York, United States
Alkeus Site
Silverdale, Washington, United States
Safety and tolerability of ALK-001 assessed by incidence and/or clinically-significant changes of a combination of ocular and non-ocular adverse events
Time frame: From baseline to 24 months
Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma
Time frame: Up to 24 months
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