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

Alkeus Pharmaceuticals

Status and phase: Active, not recruiting; Phase 2

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

Treatments: Drug: ALK-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT04239625

R01FD004098

R01FD006016 (Other Grant/Funding Number)

ALK001-P1002-EXT

R01FD006316 (U.S. FDA Grant/Contract)

Details and patient eligibility

About

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

Enrollment: 200 estimated patients

Sex: All

Ages: 8 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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200 participants in 1 patient group

ALK-001

Experimental group

Treatment:

Drug: ALK-001