This study is in progress, not accepting new patients

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

a study on Stargardt Disease Macular Degeneration

Summary

Eligibility
for people ages 8-70 (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
completion around

Description

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

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

Keywords

Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related), Macular Degeneration, Vitamin A, Retinol acetate, ALK-001

Eligibility

For people ages 8-70

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

Locations

  • Alkeus Site
    Los Angeles California 90095 United States
  • Alkeus Site
    Phoenix Arizona 85020 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alkeus Pharmaceuticals, Inc.
Links
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ID
NCT04239625
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 200 study participants
Last Updated