Optimizing busulfan dosing for infants with SCID undergoing donor stem cell transplant
Official title: Clinical and mechanistic studies defining optimal preparative approaches to infants with IL2RG/JAK3/RAG1/RAG2 SCID: a randomized trial of busulfan dosage
Funded at National Marrow Donor Program · NIH-11392252 · Data updated 2026-11-12
A study tests different busulfan doses in babies with SCID to see which helps transplant success and immune recovery.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | National Marrow Donor Program NIH-funded |
| Lab location | 1 site (Minneapolis, United States) |
| Project start | August 13, 2024 |
| Project end | May 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $958,627 |
| NIH project number | 5U01AI184132-03 |
| Project ID | NIH-11392252 on NIH RePORTER |
Who could benefit from this research
✓Could help
Infants and toddlers with SCID who are candidates for allogeneic hematopoietic cell transplantation and are receiving busulfan-based conditioning.
✕Less likely to help
Children with SCID who are not undergoing busulfan-based conditioning or who are not transplant candidates may not benefit.
What this research studies
This trial focuses on infants with severe combined immunodeficiency who need stem cell transplant. It compares low-dose versus moderate-dose busulfan conditioning to balance effective engraftment with safety. The study uses a randomized design and measures T and B cell reconstitution along with safety outcomes. It involves multiple transplant centers and includes adjuvant therapies like anti-thymocyte globulin as part of the conditioning and transplant process. If successful, the findings could guide conditioning practices to improve immune recovery after transplant in SCID patients.
Why it matters
Potential benefit: If successful, the trial could improve immune reconstitution and survival for infants with SCID undergoing transplant by identifying safer, effective conditioning.
How similar studies have gone: Earlier trials suggested that tailored busulfan dosing can balance efficacy and safety, and this study builds on that approach with a larger, multicenter randomized design.
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
Related research
Other NIH-funded projects on the same topics.
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Optimizing busulfan dosing for infants with SCID undergoing stem cell transplant
This grant tests the safest busulfan dosing levels in infants with SCID to help achieve good T and B cell recovery after a stem cell transplant.
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Providing Pediatric Transplant Access and Personalized Conditioning for Children
A national program helps kids with life-threatening blood diseases access allogeneic hematopoietic cell transplantation and tailors pre-transplant chemotherapy to each child.
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Choosing the right busulfan dose before transplant for infants with IL2RG, JAK3, or RAG1/2 SCID
This project compares two chemotherapy (busulfan) dosing strategies to help infants with certain genetic forms of SCID regain their immune cells after a stem cell transplant.
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Expanding access to bone marrow and cellular therapies for children with life‑threatening diseases
This program helps children with serious blood and genetic conditions get personalized bone marrow and cellular therapies through a network of transplant centers and trials.
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Finding the best busulfan dose for infants with SCID
This project compares lower versus moderate doses of the chemotherapy drug busulfan to help infants with IL2RG-, JAK3-, or RAG1/2-related SCID rebuild their immune systems after a donor bone marrow transplant.
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Access to clinical trials for children with life-threatening diseases through transplantation.
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.
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.