Biomarkers and clinical tools for Sanfilippo syndrome type C (MPS IIIC)
Identification of Potential Biomarkers and Clinical Tools for use in Mucopolysaccharidosis IIIC Patients.
['FUNDING_SBIR_2'] · PHOENIX NEST, INC. · NIH-11128545
Create new lab tests and clinical tools to help detect and follow MPS IIIC (Sanfilippo type C) in affected children and adults.
Quick facts
| Phase | ['FUNDING_SBIR_2'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | PHOENIX NEST, INC. (nih funded) |
| Locations | 1 site (BROOKLYN, UNITED STATES) |
| Trial ID | NIH-11128545 on ClinicalTrials.gov |
What this research studies
If you join this project, the team will work to find measurable signs in blood, urine, or other samples that link to MPS IIIC. They will collect samples and clinical information from people with confirmed or suspected MPS IIIC and compare them to controls. The researchers will develop laboratory assays and clinical tools and test whether these markers reliably track disease status or progression. Successful markers could be used to help doctors monitor symptoms and to support future treatment trials.
Who could benefit from this research
Good fit: Children, teens, or adults with confirmed or suspected MPS IIIC and caregivers willing to provide biological samples and medical history are the ideal candidates.
Not a fit: People without MPS IIIC (for example those with other types of MPS or unrelated conditions) are unlikely to receive direct benefit from these specific biomarkers.
Why it matters
Potential benefit: These tests could allow earlier diagnosis and better tracking of disease progression, helping guide care and future treatments for people with MPS IIIC.
How similar studies have performed: Some biomarker work has been done for other Sanfilippo subtypes, but validated MPS IIIC-specific clinical markers are limited, so this approach is relatively novel for this subtype.
Where this research is happening
BROOKLYN, UNITED STATES
- PHOENIX NEST, INC. — BROOKLYN, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SINGAMSETTY, SRIKANTH — PHOENIX NEST, INC.
- Study coordinator: SINGAMSETTY, SRIKANTH
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.