Access to clinical trials for children with life-threatening diseases through transplantation.
Official title: BMT Core - Pediatric Transplantation and Cellular Therapy Consortium (PTCTC): Providing Clinical Trial Access For Children With Life Threatening Diseases
Funded at National Marrow Donor Program · NIH-10939222 · Data updated 2026-11-12
This study is all about making bone marrow transplants better for kids with blood diseases by using DNA tests to personalize their treatment and find the right chemotherapy dose just for them.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | National Marrow Donor Program NIH-funded |
| Lab location | 1 site (Minneapolis, United States) |
| Project start | August 15, 2024 |
| Project end | June 30, 2031 |
| Funding year | 2024 |
| Award (this year) | $184,489 |
| NIH project number | 1UG1HL174426-01 |
| Project ID | NIH-10939222 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for participation are children aged 0-21 who have been diagnosed with life-threatening diseases that may be treated with HCT.
✕Less likely to help
Patients with conditions that are not amenable to hematopoietic cell transplantation or those outside the age range of 0-21 may not benefit from this research.
What this research studies
This research focuses on improving access to allogeneic hematopoietic cell transplantation (HCT) for children suffering from both malignant and non-malignant diseases. It aims to personalize treatment approaches based on individual patient needs, particularly for those with genetic conditions identified early in life. By utilizing DNA-based screening and diagnostic testing, the research seeks to optimize chemotherapy dosing and enhance the overall success of transplantation procedures. Patients will be evaluated for their unique immune responses and metabolic profiles to tailor their treatment effectively.
Why it matters
Potential benefit: If successful, this research could significantly improve treatment outcomes and quality of life for children with life-threatening diseases requiring transplantation.
How similar studies have gone: Previous research has shown success in personalized approaches to HCT, indicating a promising avenue for improving patient outcomes in this area.
Where this research is happening
-
National Marrow Donor Program
Minneapolis, United States Active
Researchers
- Principal investigator: Stefanski, Heather E — National Marrow Donor Program
- Study coordinator: Stefanski, Heather E
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.