ALK-001: Oral administration of a pill for up to 24 months
Study summary
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
Eligibility
Sex
ALL
Min age
8 Years
Max age
70 Years
Healthy volunteers
No
Simplified Inclusion Criteria:
* Clinical diagnosis of Stargardt disease (STGD1)
* Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor
* Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye
* 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 been invited to participate in this extension, and has signed and dated the informed consent forms (or assent where appropriate) to participate
* Female of childbearing potential has signed the attestation on contraception requirements
Simplified Exclusion Criteria:
* Is lactating or pregnant
* Has a medical condition likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or performance of study procedures
* Has abnormal laboratory result(s) at screening
* Has an ocular disorder that may confound ocular assessments
* Has a history of ocular intervention within 90 days of screening
Primary outcome measure(s)
Safety and tolerability of ALK-001 assessed by incidence and/or clinically-significant changes of a combination of ocular and non-ocular adverse events — From baseline to 24 months
Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma — Up to 24 months
Trial sites (8)
Facility
City
Region
Status
Alkeus Site
Phoenix
Arizona
Alkeus Site
Los Angeles
California
Alkeus Site
Aurora
Colorado
Alkeus Site
Gainesville
Florida
Alkeus Site
Indianapolis
Indiana
Alkeus Site
Grand Rapids
Michigan
Alkeus Site
Westbury
New York
Alkeus Site
Silverdale
Washington
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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