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

• ClinicalTrials.gov Identifier: NCT04239625
• Recruitment Status: Enrolling by invitation
• First Posted: January 27, 2020
• Last Update Posted: January 27, 2020
• Sponsor: Alkeus Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Alkeus Pharmaceuticals, Inc.

Tracking Information

• First Submitted Date: January 11, 2020
• First Posted Date: January 27, 2020
• Last Update Posted Date: January 27, 2020
• Actual Study Start Date: December 20, 2019
• Estimated Primary Completion Date: December 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 22, 2020)

• Safety and tolerability of ALK-001 assessed by incidence and/or clinically-significant changes of a combination of ocular and non-ocular adverse events [ Time Frame: From baseline to 24 months ]
• Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma [ Time Frame: Up to 24 months ]

• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

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

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

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

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

Intervention

Drug: ALK-001

Oral administration of a pill for up to 24 months

Other Names:

• C20-D3-Retinyl Acetate
• C20 Deuterated vitamin A

Study Arms

Experimental: ALK-001

Intervention: Drug: ALK-001

Publications *

• 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):7966-74. doi: 10.1074/jbc.M110.178657. Epub 2010 Dec 14.
• 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):7958-65. doi: 10.1074/jbc.M110.178640. Epub 2010 Nov 12.
• 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.
• 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.
• 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.

Recruitment Information

• Recruitment Status: Enrolling by invitation
• Estimated Enrollment (submitted: January 22, 2020): 140
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 2024
• Estimated Primary Completion Date: December 2024 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 8 Years to 70 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04239625
• Other Study ID Numbers: ALK001-P1002-EXT; R01FD004098 ( U.S. FDA Grant/Contract ); R01FD006016 ( Other Grant/Funding Number: FDA OOPD )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Responsible Party: Alkeus Pharmaceuticals, Inc.
• Study Sponsor: Alkeus Pharmaceuticals, Inc.
• Collaborators: Not Provided

Investigators

• Study Director: Leonide Saad, PhD; Alkeus Pharmaceuticals, Inc.

• PRS Account: Alkeus Pharmaceuticals, Inc.
• Verification Date: January 2020