Investigating a new oral treatment for Stargardt disease to slow vision loss.
ALK-001 Phase 2 Treatment of Stargardt Disease IND 108,353 (09/08/2015)
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 ID | NIH-10433821 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Stargardt disease who are experiencing vision loss.
Not a fit: Patients with other forms of vision impairment unrelated to Stargardt disease may not benefit from this research.
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 performed: 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
Somerville, United States
- Alkeus Pharmaceuticals, INC. — Somerville, United States (Active)
Researchers
- Principal investigator: Saad, Leonide — Alkeus Pharmaceuticals, INC.
- Study coordinator: Saad, Leonide
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 below.