Trials / Enrolling By Invitation
Enrolling By InvitationNCT02402660
Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease
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
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 160 (estimated)
- Sponsor
- Alkeus Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- 8 Years – 70 Years
- Healthy volunteers
- Not accepted
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
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.
Conditions
- Stargardt Disease
- Stargardt Macular Degeneration
- Stargardt Macular Dystrophy
- Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | ALK-001 | Daily, oral administration for 24 months |
| DRUG | Placebo | Daily, oral administration for 24 months |
Timeline
- Start date
- 2015-08-01
- Primary completion
- 2026-06-30
- Completion
- 2026-11-30
- First posted
- 2015-03-30
- Last updated
- 2025-04-27
Locations
14 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT02402660. Inclusion in this directory is not an endorsement.