Oral N‑acetylcysteine treatment for retinitis pigmentosa
Official title: NAC Attack, a phase-3, multicenter, randomized, placebo-controlled trial in patients with retinitis pigmentosa
Funded at Johns Hopkins University · NIH-11179132 · Data updated 2026-11-12
Adults with retinitis pigmentosa will take oral N‑acetylcysteine or a placebo to see whether it slows vision loss.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | March 17, 2022 |
| Project end | July 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $3,183,918 |
| NIH project number | 5UG1EY033293-04 |
| Project ID | NIH-11179132 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with a confirmed diagnosis of retinitis pigmentosa who meet the study's eligibility criteria and can take oral medication and attend regular visits are the intended participants.
✕Less likely to help
People without retinitis pigmentosa, those with very advanced end‑stage retinal loss, or anyone with medical reasons not to take N‑acetylcysteine are unlikely to benefit from joining.
What this research studies
You would be randomly assigned to take oral N‑acetylcysteine (1800 mg twice daily) or a placebo in a double‑masked way, with more people receiving the active pill than placebo (2:1). The trial runs at multiple clinical sites and follows participants for about 45 months with regular eye exams and testing. Johns Hopkins serves as the coordinating center to manage sites, data, and quality assurance. The goal is to track changes in vision and retinal health over time to determine whether the medicine can slow degeneration.
Why it matters
Potential benefit: If effective, the treatment could slow or prevent further vision loss in people with retinitis pigmentosa.
How similar studies have gone: Smaller clinical studies and laboratory research have suggested NAC may protect photoreceptors, but this larger phase‑3 trial is designed to provide definitive evidence.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Kong, Xiangrong — Johns Hopkins University
- Study coordinator: Kong, Xiangrong
Related research
Other NIH-funded projects on the same topics.
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This grant studies how specific retinal extracellular matrix proteins (IMPG1 and IMPG2) affect eye health and contribute to retinitis pigmentosa, with potential implications for people who have or are at risk for inherited blindness.
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NAC Attack: a late-stage trial for preserving vision in RP patients
A large, multi-site study is testing whether taking a daily NAC pill can help people with retinitis pigmentosa keep more of their vision.
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Non-invasive biomarker for mitochondria health in retinitis pigmentosa
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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.