Testing a treatment to help preserve vision in patients with retinitis pigmentosa
Official title: NAC Attack, A Phase-3, Multicenter, Randomized, Placebo-Controlled Trial in Patents with Retinitis Pigmentosa
Funded at Johns Hopkins University · NIH-11045045 · Data updated 2026-11-12
This study is looking at whether taking N-acetylcysteine (NAC), a helpful antioxidant, can support vision in people with retinitis pigmentosa by protecting important cells in the eye, and participants will either receive NAC or a placebo over time to see how it affects their eyesight.
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 | February 28, 2027 |
| Funding year | 2025 |
| Award (this year) | $302,263 |
| NIH project number | 5UG1EY033286-04 |
| Project ID | NIH-11045045 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with retinitis pigmentosa who are experiencing vision loss.
✕Less likely to help
Patients with other forms of retinal degeneration or those who do not have retinitis pigmentosa may not benefit from this research.
What this research studies
This research investigates the effects of N-acetylcysteine (NAC), an antioxidant, on patients with retinitis pigmentosa (RP), a condition that leads to severe visual impairment. The study involves a multicenter, randomized, double-masked, placebo-controlled trial where participants will receive either NAC or a placebo over an extended period. The primary goal is to determine if long-term NAC administration can promote the survival of cone photoreceptors, which are crucial for vision. Patients will be monitored using advanced imaging techniques to assess changes in their retinal health and visual function.
Why it matters
Potential benefit: If successful, this research could lead to a new treatment that helps preserve vision and improve quality of life for patients with retinitis pigmentosa.
How similar studies have gone: Previous studies have shown promising results with NAC in animal models and small clinical trials, suggesting potential for success in this larger trial.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Campochiaro, Peter a — Johns Hopkins University
- Study coordinator: Campochiaro, Peter a
Related research
Other NIH-funded projects on the same topics.
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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.