Overview

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

Status:
Enrolling by invitation
Trial end date:
2024-12-01
Target enrollment:
0
Participant gender:
All
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
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Alkeus Pharmaceuticals, Inc.
Treatments:
Retinol acetate
Vitamin A
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