Autosomal Recessive Stargardt Disease 1 (ABCA4-related), Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
Conditions
Brief 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
Interventions
DRUGALK-001
Oral administration of a pill for up to 24 months
Sponsors
Alkeus Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
8 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 |
Countries
United States
Outcome results
None listed