Developing a new treatment for Infantile Neuronal Ceroid Lipofuscinosis (INCL)
Official title: Development of N-tert-(Butyl)hydroxylamine (NtBuHA) as a therapeutic agent for treating Infantile Neuronal Ceroid Lipofuscinosis (INCL)
Funded at Circumvent Pharmaceuticals, INC. · NIH-10490336 · Data updated 2026-11-12
This study is testing a new treatment called NtBuHA for children with Infantile Neuronal Ceroid Lipofuscinosis (INCL), a serious brain disorder, to see if it can help improve their health and quality of life.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Circumvent Pharmaceuticals, INC. NIH-funded |
| Lab location | 1 site (Portland, United States) |
| Project start | September 20, 2021 |
| Project end | June 30, 2025 |
| Funding year | 2022 |
| Award (this year) | $1,449,910 |
| NIH project number | 5R44NS120360-02 |
| Project ID | NIH-10490336 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with Infantile Neuronal Ceroid Lipofuscinosis (INCL), particularly those under the age of 11.
✕Less likely to help
Patients with other forms of Batten disease or those not diagnosed with INCL may not benefit from this research.
What this research studies
This research focuses on creating a new therapeutic agent, N-tert-(Butyl)hydroxylamine (NtBuHA), to treat Infantile Neuronal Ceroid Lipofuscinosis (INCL), a rare and severe neurological disorder in children. The team at Circumvent Pharmaceuticals is conducting essential studies to ensure the safety and effectiveness of this treatment, including toxicity assessments and formulation development. The goal is to provide a viable treatment option that addresses the underlying causes of INCL, moving beyond just supportive care. If successful, this could significantly improve the quality of life for affected children.
Why it matters
Potential benefit: If successful, this research could provide the first effective treatment option for children suffering from INCL.
How similar studies have gone: While there is a significant unmet need for treatments for INCL, this specific approach using NtBuHA is novel and has not been tested in clinical settings before.
Where this research is happening
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Circumvent Pharmaceuticals, INC.
Portland, United States Active
Researchers
- Principal investigator: Chelsky, Daniel — Circumvent Pharmaceuticals, INC.
- Study coordinator: Chelsky, Daniel
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.