Batten disease natural history and trial readiness across multiple gene forms
Official title: Research Project 1
Funded at Hugo W. Moser Res Inst Kennedy Krieger · NIH-11417532 · Data updated 2026-11-12
This grant supports a large, multi-site study to understand how Batten disease progresses in people and to prepare for future clinical trials.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Hugo W. Moser Res Inst Kennedy Krieger NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | September 15, 2025 |
| Project end | August 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $799,137 |
| NIH project number | 5U54HD122210-02 |
| Project ID | NIH-11417532 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals diagnosed with neuronal ceroid lipofuscinoses (Batten diseases), especially CLN1, CLN2, CLN3, and CLN6 forms, across age ranges.
✕Less likely to help
The study may not directly benefit participants who do not have Batten disease or who cannot participate in long-term follow-up.
What this research studies
This project will collect and analyze detailed information from individuals with Batten diseases (including CLN1, CLN2, CLN3, and CLN6) to map how the illness changes over time. It aims to create patient-centered, responsive outcome measures so future therapies can be tested more efficiently. The study involves multiple sites and follows participants over time to capture natural history data that can guide trial design and regulatory decisions. The goal is to remove barriers to therapy development for these rare, devastating conditions.
Why it matters
Potential benefit: If successful, patients could see faster, better-designed clinical trials and more effective treatments become available sooner.
How similar studies have gone: This is the first large multi-site natural history collaboration for NCLs in the United States, building on prior single-site efforts.
Where this research is happening
-
Hugo W. Moser Res Inst Kennedy Krieger
Baltimore, United States Active
Researchers
- Principal investigator: Augustine, Erika — Hugo W. Moser Res Inst Kennedy Krieger
- Study coordinator: Augustine, Erika
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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.