New treatment approach for a common kidney disease.
Developing a New Therapeutic Approach for Autosomal Dominant Polycystic Kidney Disease.
This study is looking at whether a drug called VX-809, which is used for cystic fibrosis, can help reduce kidney cysts and improve kidney function in people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), giving patients a new treatment option.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project ID | NIH-10836990 on NIH RePORTER |
What this research studies
This research focuses on developing a new therapeutic approach for Autosomal Dominant Polycystic Kidney Disease (ADPKD), a genetic disorder that leads to kidney failure in many patients. The study investigates the use of VX-809, a drug currently used for cystic fibrosis, to see if it can reduce cyst growth and improve kidney function in ADPKD patients. By leveraging existing clinical data, the research aims to fast-track this treatment for ADPKD, potentially offering a new option for patients who currently have limited treatment choices.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with Autosomal Dominant Polycystic Kidney Disease.
Not a fit: Patients with other forms of kidney disease or those who do not have ADPKD may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a new effective treatment option for patients with ADPKD, potentially delaying the need for kidney transplants.
How similar studies have performed: Previous research has shown promise in using CFTR modulators for similar conditions, indicating potential success for this novel application.
Where this research is happening
Baltimore, United States
- Johns Hopkins University — Baltimore, United States (Active)
Researchers
- Principal investigator: Cebotaru, Liudmila — Johns Hopkins University
- Study coordinator: Cebotaru, Liudmila
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.