Ignite Creation Date:
2025-12-25 @ 2:14 AM
Ignite Modification Date:
2026-01-08 @ 5:07 AM
Study NCT ID:
NCT02402660
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-04-27
First Post:
2015-03-09
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease
Sponsor:
Alkeus Pharmaceuticals, Inc.
Organization:
Study Overview
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
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2025-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
TEASE
Brief 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
Funding Source - FDA OOPD
Detailed Description:
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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| R01FD004098 | FDA | None | https://reporter.nih.gov/quic… | View |
| R01FD006016 | OTHER_GRANT | FDA OOPD | View | |
| R01FD006316 | FDA | None | https://reporter.nih.gov/quic… | View |