N-acetylcysteine (NAC) to help preserve vision in retinitis pigmentosa
NAC Attack, A Phase-3, Multicenter, Randomized, Placebo-Controlled Trial in Patents with Retinitis Pigmentosa
This trial tests whether taking oral NAC daily can slow cone loss and help people with retinitis pigmentosa keep more vision.
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 ID | NIH-11307042 on NIH RePORTER |
What this research studies
This is a phase 3, multicenter, randomized, double-masked trial comparing long-term oral NAC to a placebo in people with retinitis pigmentosa. Participants are randomly assigned to receive NAC or placebo, take study medication (previous work used up to 1800 mg twice daily), and come to regular clinic visits for eye imaging and vision tests. The main outcome is cone survival measured by ellipsoid zone width on OCT scans, with other functional vision measures tracked over time. About 438 participants will be enrolled across multiple centers to see if the benefits seen in animals and a small earlier human trial hold up in a larger group.
Who could benefit from this research
Good fit: People diagnosed with retinitis pigmentosa who still have measurable cone structure or function, can take oral medication, and can attend regular study visits are the likely candidates.
Not a fit: People with very advanced disease and little remaining cone tissue, or those with medical reasons they cannot take NAC, are less likely to benefit from this trial.
Why it matters
Potential benefit: If successful, long-term NAC could slow cone degeneration and help preserve visual field and acuity for people with retinitis pigmentosa.
How similar studies have performed: Animal models showed strong cone protection with antioxidants and a small 30-patient human trial found NAC was safe and produced modest short-term cone-function improvements, so this larger trial follows promising early data.
Where this research is happening
Baltimore, United States
- Johns Hopkins University — Baltimore, United States (Active)
Researchers
- Principal investigator: Campochiaro, Peter a — Johns Hopkins University
- Study coordinator: Campochiaro, Peter a
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.