Improving clinical trials for inherited brain diseases.
Official title: Global Leukodystrophy Initiative Clinical Trials Network Gap Year Administrative Supplement
Funded at Children's Hosp of Philadelphia · NIH-11084865 · Data updated 2026-11-12
This study is working to make it easier to test new treatments for leukodystrophies by teaming up with support groups to find better ways to measure how patients are doing and using medical records to gather important health information.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | July 1, 2024 |
| Project end | June 30, 2025 |
| Funding year | 2024 |
| Award (this year) | $1,009,720 |
| NIH project number | 3U54NS115052-05S4 |
| Project ID | NIH-11084865 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with specific leukodystrophies who are interested in participating in clinical trials.
✕Less likely to help
Patients with leukodystrophies that are not part of the core focus of this initiative may not receive direct benefits from this research.
What this research studies
This research focuses on enhancing the readiness for clinical trials targeting leukodystrophies, which are inherited disorders affecting the brain's white matter. By collaborating with advocacy groups, the initiative aims to develop reliable methods for assessing clinical outcomes and patient-reported experiences. The project will also utilize advanced techniques to extract valuable natural history data from electronic medical records. This comprehensive approach seeks to ensure that promising treatments can be effectively tested and brought to patients.
Why it matters
Potential benefit: If successful, this research could lead to more effective clinical trials and ultimately better treatment options for patients with leukodystrophies.
How similar studies have gone: Previous research initiatives focused on leukodystrophies have shown promise in improving clinical trial methodologies, indicating a potential for success in this approach.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Vanderver, Adeline Lucie — Children's Hosp of Philadelphia
- Study coordinator: Vanderver, Adeline Lucie
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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.