Investigating a new oral treatment for Stargardt disease to slow vision loss.
Official title: ALK-001 Phase 2 Treatment of Stargardt Disease IND 108,353 (09/08/2015)
Funded at Alkeus Pharmaceuticals, INC. · NIH-10433821 · Data updated 2026-11-12
This study is testing a new pill called ALK-001 that aims to help people with Stargardt disease by slowing down vision loss caused by a problem with a specific gene.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Alkeus Pharmaceuticals, INC. NIH-funded |
| Lab location | 1 site (Somerville, United States) |
| Project start | July 1, 2018 |
| Project end | June 30, 2025 |
| Funding year | 2023 |
| Award (this year) | $500,000 |
| NIH project number | 5R01FD006316-04 |
| Project ID | NIH-10433821 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Stargardt disease who are experiencing vision loss.
✕Less likely to help
Patients with other forms of vision impairment unrelated to Stargardt disease may not benefit from this research.
What this research studies
This research focuses on developing an oral medication called ALK-001 aimed at preventing or slowing the progression of Stargardt disease, a rare form of juvenile blindness. The study is based on the understanding that mutations in the ABCA4 gene lead to harmful vitamin A aggregates that cause vision loss. By inhibiting the formation of these aggregates, ALK-001 has shown promise in preclinical models and early clinical trials, demonstrating safety and bioactivity. The ongoing Phase 2 trial will further assess the safety and efficacy of this treatment in patients with Stargardt disease.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option that slows down vision loss for patients with Stargardt disease.
How similar studies have gone: Previous research has shown promising results with similar approaches in animal models, but this Phase 2 trial is crucial for further validation in humans.
Where this research is happening
-
Alkeus Pharmaceuticals, INC.
Somerville, United States Active
Researchers
- Principal investigator: Saad, Leonide — Alkeus Pharmaceuticals, INC.
- Study coordinator: Saad, Leonide
Related research
Other NIH-funded projects on the same topics.
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Combination drug approach to slow vision loss in Stargardt disease
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-
New treatment approach for Stargardt disease
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-
ASP2020 eye injection safety in Stargardt-type macular dystrophy
A small study will test whether a single ASP2020 eye injection is safe and may help people with Stargardt-type macular dystrophy.
-
C.001 for retinal degeneration: a first-in-human dose-escalation safety test
A first-in-human study will test the safety and tolerability of a single subretinal injection of C.001 in adults with retinal diseases involving retinal pigment epithelium degeneration.
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Functional visual field and navigation testing for moderate-to-profound inherited retinal disease
This project will test whether functional visual field and navigation tests can measure real-world vision and mobility in people with moderate-to-profound inherited retinal diseases.
-
Single intravitreal RTx-021 injection for Stargardt disease
This will test whether a single injection of RTx-021 into one eye is safe and may help people aged 16 and older with Stargardt disease.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.