Testing a new treatment for retinitis pigmentosa using N-acetylcysteine
Official title: NAC Attack, a phase-3, multicenter, randomized, placebo-controlled trial in patients with retinitis pigmentosa
Funded at Johns Hopkins University · NIH-10868502 · Data updated 2026-11-12
This study is looking at whether a supplement called N-acetylcysteine (NAC) can help slow down vision loss in people with retinitis pigmentosa, and if you join, you’ll either get NAC or a placebo for about three and a half years to see how it works.
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 | 2024 |
| Award (this year) | $2,945,503 |
| NIH project number | 5UG1EY033293-03 |
| Project ID | NIH-10868502 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals aged 20-60 who have been diagnosed with retinitis pigmentosa.
✕Less likely to help
Patients with other forms of retinal degeneration or those outside the specified age range may not benefit from this research.
What this research studies
This research investigates the effectiveness and safety of N-acetylcysteine (NAC) in treating retinitis pigmentosa, a genetic condition that leads to severe vision loss and blindness. Participants will be randomly assigned to receive either NAC or a placebo for a duration of 45 months. The study aims to determine if NAC can slow down the progression of vision loss caused by the degeneration of photoreceptors in the retina. The trial is conducted across multiple centers to ensure a diverse participant pool and robust data collection.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option to slow or prevent vision loss in patients with retinitis pigmentosa.
How similar studies have gone: While the use of N-acetylcysteine in this context is novel, previous studies have shown promise in using similar antioxidant therapies for retinal conditions.
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.
-
Inflammation in MERTK-related retinitis pigmentosa
This project aims to understand whether losing the MERTK gene causes retinal inflammation that leads to vision loss in people with MERTK-related retinitis pigmentosa.
-
Enhancing cone survival in RP through targeted cell-specific CRISPR therapy
A patient-friendly look at a new approach to protect cone cells in retinitis pigmentosa using precise gene editing to support vision.
-
Oral, once-daily MC16 medicine for retinitis pigmentosa
A gene-mutation-agnostic pill (MC16) is being studied to help people with retinitis pigmentosa by addressing a common cellular problem in the eye.
-
Role of IPM Proteoglycans in Retinal Health and Inherited Blindness
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.
-
Non-invasive biomarker for mitochondria health in retinitis pigmentosa
Aims to test a new, non-invasive OCT-based biomarker to explain and predict how mitochondria function affects cone cells in people with retinitis pigmentosa.
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.