Stargardt Disease Clinical Trial
Official title:
A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease
Verified date | June 2024 |
Source | Alkeus Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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
Status | Enrolling by invitation |
Enrollment | 140 |
Est. completion date | December 2026 |
Est. primary completion date | December 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 8 Years to 70 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Coordinating Center | Somerville | Massachusetts |
Lead Sponsor | Collaborator |
---|---|
Alkeus Pharmaceuticals, Inc. |
United States,
Charbel Issa P, Barnard AR, Herrmann P, Washington I, MacLaren RE. Rescue of the Stargardt phenotype in Abca4 knockout mice through inhibition of vitamin A dimerization. Proc Natl Acad Sci U S A. 2015 Jul 7;112(27):8415-20. doi: 10.1073/pnas.1506960112. Epub 2015 Jun 23. — View Citation
Kaufman Y, Ma L, Washington I. Deuterium enrichment of vitamin A at the C20 position slows the formation of detrimental vitamin A dimers in wild-type rodents. J Biol Chem. 2011 Mar 11;286(10):7958-7965. doi: 10.1074/jbc.M110.178640. Epub 2010 Nov 12. — View Citation
Ma L, Kaufman Y, Zhang J, Washington I. C20-D3-vitamin A slows lipofuscin accumulation and electrophysiological retinal degeneration in a mouse model of Stargardt disease. J Biol Chem. 2011 Mar 11;286(10):7966-7974. doi: 10.1074/jbc.M110.178657. Epub 2010 Dec 14. — View Citation
Mihai DM, Jiang H, Blaner WS, Romanov A, Washington I. The retina rapidly incorporates ingested C20-D(3)-vitamin A in a swine model. Mol Vis. 2013 Jul 25;19:1677-83. Print 2013. — View Citation
Saad L, Washington I. Can Vitamin A be Improved to Prevent Blindness due to Age-Related Macular Degeneration, Stargardt Disease and Other Retinal Dystrophies? Adv Exp Med Biol. 2016;854:355-61. doi: 10.1007/978-3-319-17121-0_47. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | ||
Primary | Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma | Up to 24 months |
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