Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04239625
• Other study ID #: ALK001-P1002-EXT
• Secondary ID: R01FD004098R01FD
• Status: Enrolling by invitation
• Phase: Phase 2
• Start date: December 20, 2019
• Est. completion date: December 2026

Study information

• Verified date: January 2024
• Source: Alkeus Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• 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

Related Conditions & MeSH terms

• Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
• Macular Degeneration
• Stargardt Disease
• Stargardt Macular Degeneration
• Stargardt Macular Dystrophy

Intervention

Drug:
• ALK-001
Oral administration of a pill for up to 24 months

Locations

Country	Name	City	State
United States	Coordinating Center	Somerville	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Alkeus Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

References & Publications (5)

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

Outcome

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