Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
Study Details
Study Description
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
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: ALK-001
|
Drug: ALK-001
Oral administration of a pill for up to 24 months
Other Names:
|
Outcome Measures
Primary Outcome Measures
- 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]
Eligibility Criteria
Criteria
Simplified Inclusion Criteria:
-
Clinical diagnosis of Stargardt disease (STGD1)
-
Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor
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Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye
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Healthy as judged by investigator
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Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study
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Has been invited to participate in this extension, and has signed and dated the informed consent forms (or assent where appropriate) to participate
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Female of childbearing potential has signed the attestation on contraception requirements
Simplified Exclusion Criteria:
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Is lactating or pregnant
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Has a medical condition likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or performance of study procedures
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Has abnormal laboratory result(s) at screening
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Has an ocular disorder that may confound ocular assessments
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Has a history of ocular intervention within 90 days of screening
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Coordinating Center | Somerville | Massachusetts | United States | 02144 |
Sponsors and Collaborators
- Alkeus Pharmaceuticals, Inc.
Investigators
- Study Director: Leonide Saad, PhD, Alkeus Pharmaceuticals, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
- 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.
- 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.
- 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.
- Mihai DM, Jiang H, Blaner WS, Romanov A, Washington I. The retina rapidly incorporates ingested C20-D₃-vitamin A in a swine model. Mol Vis. 2013 Jul 25;19:1677-83. Print 2013.
- 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.
- ALK001-P1002-EXT
- R01FD004098
- R01FD006016