Developing new drugs for CLN3 and CLN6 diseases in children
Small Molecule Drug Discovery for CLN3 and CLN6 Disease
['FUNDING_R01'] · UNIVERSITY OF NEBRASKA MEDICAL CENTER · NIH-10873979
This study is looking for new medicines to help kids with CLN3 and CLN6 diseases by understanding how their cells work, with the hope of finding better ways to slow down the disease and make life easier for them.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF NEBRASKA MEDICAL CENTER (nih funded) |
| Locations | 1 site (OMAHA, UNITED STATES) |
| Trial ID | NIH-10873979 on ClinicalTrials.gov |
What this research studies
This research focuses on finding new small molecule drugs to treat CLN3 and CLN6 diseases, which are severe neurodegenerative disorders affecting children. The approach involves understanding the underlying cellular processes, such as autophagy and apoptosis, that contribute to the progression of these diseases. By targeting these processes simultaneously, the researchers aim to develop effective treatments that can modify the disease course and improve the quality of life for affected children. The study is particularly urgent as there are currently no effective treatments available for these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with CLN3 or CLN6 diseases, typically under the age of 11.
Not a fit: Patients with other forms of neurodegenerative diseases not related to CLN3 or CLN6 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve outcomes for children suffering from CLN3 and CLN6 diseases.
How similar studies have performed: While the approach of targeting multiple pathways in neurodegenerative diseases is relatively novel, similar strategies have shown promise in cancer treatment.
Where this research is happening
OMAHA, UNITED STATES
- UNIVERSITY OF NEBRASKA MEDICAL CENTER — OMAHA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: TRIPPIER, PAUL — UNIVERSITY OF NEBRASKA MEDICAL CENTER
- Study coordinator: TRIPPIER, PAUL
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.
Conditions: Batten Disease