Biomarkers to monitor Niemann-Pick Type C
Clinically Relevant Biomarkers for Niemann-Pick Type C
['FUNDING_U01'] · UNIVERSITY OF ILLINOIS AT CHICAGO · NIH-11162466
This project looks at blood and tissue markers to help track disease progress and treatment response in people with Niemann-Pick Type C.
Quick facts
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF ILLINOIS AT CHICAGO (nih funded) |
| Locations | 1 site (Chicago, UNITED STATES) |
| Trial ID | NIH-11162466 on ClinicalTrials.gov |
What this research studies
If you have Niemann-Pick Type C, the team will use samples from a natural history study to test a set of candidate biomarkers. They will prioritize and validate markers that reflect brain changes and other disease pathways using custom laboratory methods that can be shared with other centers. The work includes analyzing twelve candidate markers in a larger group of biospecimens to see which ones predict progression and response to therapy. The goal is to use these markers to give better prognoses, help with family counseling, and group patients for future clinical trials.
Who could benefit from this research
Good fit: People with a confirmed diagnosis of Niemann-Pick Type C (especially NPC1) who can provide blood, cerebrospinal fluid, or tissue samples and join a natural history or biospecimen study.
Not a fit: Unaffected carriers or people without a confirmed NPC diagnosis, and those unable to provide samples, are unlikely to get direct benefit from this project.
Why it matters
Potential benefit: If successful, these biomarkers could let doctors monitor disease, predict outcomes, and pick the right patients for clinical trials.
How similar studies have performed: Other natural history and biomarker studies in NPC have identified promising candidates but none are yet established as standard trial endpoints, so this work builds on prior progress.
Where this research is happening
Chicago, UNITED STATES
- UNIVERSITY OF ILLINOIS AT CHICAGO — Chicago, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: COLOGNA, STEPHANIE M — UNIVERSITY OF ILLINOIS AT CHICAGO
- Study coordinator: COLOGNA, STEPHANIE M
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.