Summary

Eligibility
for people ages 8-70 (full criteria)
Location
at UCLA
Dates
study started
completion around
Principal Investigator
by Michael Gorin, MD, PhD (ucla)

Description

Summary

The purpose of this study is to determine the long term safety and tolerability of ALK-001 (C20-D3-retinyl acetate), and to explore the effects of ALK-001 on the progression of Stargardt disease in patients between the ages of 8 and 70 years old.

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

Details

This study evaluates the effects of orally-administered ALK-001 on the progression of Stargardt disease (ABCA4-related). Stargardt disease is a rare genetic disorder that leads to damage to the retina and results in legal blindness. Stargardt disease is caused by a defective ABCA4 gene, which affects the processing of vitamin A in the eye and leads to the formation of toxic vitamin A aggregates (called "vitamin A dimers") in the eye. Vitamin A dimers are thought to contribute to vision loss in Stargardt disease. ALK-001, the investigational drug, is a chemically-modified vitamin A designed as a replacement of vitamin A to prevent the formation of toxic vitamin A dimers in the eye. Trial participants will receive either ALK-001 or placebo, and follow-up visits will take place periodically for up to 24 months. There is currently no treatment for 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

Locations

  • University of California Los Angeles - Jules Stein Eye Institute accepting new patients
    Los Angeles California 90095 United States
  • University of Utah - Moran Eye Institute completed
    Salt Lake City Utah 84132 United States
  • Vitreoretinal Associates accepting new patients
    Gainesville Florida 32607 United States

Lead Scientist at University of California Health

  • Michael Gorin, MD, PhD (ucla)
    Michael B. Gorin, M.D., Ph.D., holds the Harold and Pauline Price Chair in Ophthalmology.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alkeus Pharmaceuticals, Inc.
ID
NCT02402660
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 140 study participants
Last Updated