Ignite Creation Date:
2024-05-06 @ 3:55 AM
Last Modification Date:
2024-10-26 @ 11:40 AM
Study NCT ID:
NCT02402660
Status:
RECRUITING
Last Update Posted:
2024-06-21
First Post:
2015-03-09
Brief Title:
Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease
Sponsor:
Alkeus Pharmaceuticals Inc
Organization:
Alkeus Pharmaceuticals Inc
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:
RECRUITING
Status Verified Date:
2024-06
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:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| R01FD006016 | OTHER_GRANT | FDA OOPD | httpsreporternihgovquickSearchR01FD004098 |
| R01FD004098 | FDA | None | None |
| R01FD006316 | FDA | None | None |