Data Management and Coordination for Rare Disease Research Network
Official title: Data Management and Coordinating Center: Rare Diseases Clinical Research Network
Funded at Cincinnati Childrens Hosp Med Ctr · NIH-11360146 · Data updated 2026-11-12
A national effort to organize and manage information for rare disease studies to help doctors and researchers learn more and speed up treatments for children with rare conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project start | August 5, 2019 |
| Project end | June 30, 2030 |
| Funding year | 2026 |
| Award (this year) | $5,541,569 |
| NIH project number | 5U2CTR002818-07 |
| Project ID | NIH-11360146 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with suspected or confirmed rare diseases who may participate in future RDCRN studies or benefit from improved care pathways.
✕Less likely to help
Some patients may not directly participate in trials or see immediate treatment changes during the project period.
What this research studies
This grant funds a central data and coordination center that supports many rare disease studies focused on children. It provides expert help in study design, data management, biostatistics, and epidemiology to ensure high-quality evidence can be built from patient information and research results. By standardizing definitions and improving data sharing across sites, it aims to accelerate clinical trials and safer, more effective treatments for kids with rare diseases. The work relies on collaboration with a network of Rare Diseases Clinical Research Consortia to improve diagnosis, care, and outcomes for affected families.
Why it matters
Potential benefit: If successful, families affected by rare diseases could see faster access to better diagnostics, care plans, and potential new therapies.
How similar studies have gone: Previous RDCRN efforts have shown that centralized data management can improve trial readiness and study quality in rare diseases.
Where this research is happening
-
Cincinnati Childrens Hosp Med Ctr
Cincinnati, United States Active
Researchers
- Principal investigator: Macaluso, Maurizio — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Macaluso, Maurizio
Related research
Other NIH-funded projects on the same topics.
-
Liver pathobiology from rare liver diseases
A study at UCSF looks at why rare liver conditions cause liver damage, with the aim of finding new ways to diagnose and treat chronic liver disease.
-
Data Management and Coordinating Center support for Rare Diseases Research
A national rare diseases research program is funding a center to manage data and coordinate studies to help improve diagnosis, care, and potential treatments for children with rare conditions.
-
EEG-based drug repurposing for rare CNS diseases
A new approach uses EEG brain signatures to find existing drugs that might help people with rare nervous-system disorders.
-
Ethics of Engaging Patient Groups in Drug Development for Rare Diseases
This grant asks how patient and family groups can ethically help shape rare-disease drug development from idea to market, with input from patients, families, doctors, and researchers.
-
Center for Polyamine Disorders group study of rare metabolic disorders
A research consortium is studying a group of rare metabolic disorders linked to polyamines and aims to understand symptoms, biomarkers, and disease progression to improve care.
-
Encore: speeding up finding new uses for existing drugs to help kids with rare diseases
A big NIH project will test thousands of existing medicines to see if any can help treat rare genetic diseases in children.
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.