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

NCT04239625 | Active Not Recruiting Phase 2 FDA Drug

• 4 other identifiers: ALK001-P1002-EXTR01FD004098R01FD006016R01FD006316
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 8

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

Conditions

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

Outcome Measures

Primary Outcomes (2)

• 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

Study Arms (1)

ALK-001

EXPERIMENTAL

Drug: ALK-001

Interventions

ALK-001 DRUG

Oral administration of a pill for up to 24 months

Also known as: C20-D3-Retinyl Acetate, C20 Deuterated vitamin A

ALK-001

Eligibility Criteria

• Age: 8 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alkeus Pharmaceuticals, Inc.: lead

Study Sites (8)

Alkeus Site

Phoenix, Arizona, 85020, United States

Location

Alkeus Site

Los Angeles, California, 90095, United States

Location

Alkeus Site

Aurora, Colorado, 80045, United States

Location

Alkeus Site

Gainesville, Florida, 32601, United States

Location

Alkeus Site

Indianapolis, Indiana, 46202, United States

Location

Alkeus Site

Grand Rapids, Michigan, 49456, United States

Location

Alkeus Site

Westbury, New York, 11590, United States

Location

Alkeus Site

Silverdale, Washington, 98383, United States

Location

Related Publications (5)

• 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.

  PMID: 21156790 BACKGROUND

• 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.

  PMID: 21075840 BACKGROUND

• 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.

  PMID: 23914132 BACKGROUND

• 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.

  PMID: 26106163 BACKGROUND

• 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.

  PMID: 26427432 BACKGROUND

Related Links

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MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 11, 2020
• First Posted: January 27, 2020
• Study Start: December 20, 2019
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: April 27, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)