# Blood and Marrow Transplant Clinical Trials Network

> **NIH NIH UG1** · DUKE UNIVERSITY · 2022 · $175,129

## Abstract

Abstract
The pediatric and adult transplant programs at Duke University Medical Center have served as a core clinical
center for the BMT-CTN since its establishment in 2001. As a core center, Duke has made significant
contributions to the network, including enrollment of 310 patients on CTN protocols, chairing two protocols (Dr.
Kurtzberg chaired 0501 and Dr. Horwitz chaired 0901); serving on 6 protocol committees and 2 disease
committees, authoring manuscripts and establishing and managing the immune reconstitution core for protocol
0501. The center has been in compliance with accurate and timely data and bio-specimen submission. The PI
and Co-PI have regularly attended steering committee meetings, conference calls and the SSOS meetings.
For this renewal of the BMT-CTN, Duke brings three specialized types of expertise to the table, (1) a long-
standing and pioneering role in cord blood transplantation and banking and (2) unique experience in the
treatment of pediatric patients with hematopoietic stem cell transplantation for non-malignant diseases and (3)
pioneering work in cord blood and cord-tissue derived cellular therapies. In this application, Duke has added
two additional centers as part of a Duke Consortium to increase participation in the network and also to
increase accrual to BMT-CTN trials.
The study concept proposed in this application, will serve as the foundation for a follow-on study to BMT-CTN
0501. The clinical trial proposed will compare in a randomized phase II clinical trial, the outcomes of related
haplo-identical bone marrow transplantation or unrelated donor cord blood transplantation in children with high
risk leukemias undergoing myeloablative conditioning therapy. The supportive care will be standardized. The
preparative regimens and GvHD prophylaxis will be based on treatment `packages' well studied for the cord
blood group and piloted for the haplo-BMT group. They are standardized to the extent possible, with the
splitting cyclophosphamide for pre and post-transplant dosing for the haplo group and use of fludarabine in the
cord blood group. The primary endpoint will be relapse-free survival at 2 years post-transplant. Overall survival,
engraftment, acute and chronic Graft-versus-Host Disease, treatment related mortality, immune reconstitution,
and transplant feasibility will also be explored as secondary endpoints. The predictive role of MRD at the time
of transplant will also be examined to determine if either graft source offers better protection against post-
transplant leukemic relapse. This study will answer critical and timely questions about the role of various
alternative donors and identification of best practices for alternative donor selection for transplantation of
children with hematological malignancies lacking matched related or unrelated donors.
We plan to fund this trial by responding to the FOA Clinical Coordinating Center for Multi-Site Investigator-
Initiated Clinical Trials (Collaborative UG3/UH3)...

## Key facts

- **NIH application ID:** 10429979
- **Project number:** 5UG1HL069274-22
- **Recipient organization:** DUKE UNIVERSITY
- **Principal Investigator:** MITCHELL E HORWITZ
- **Activity code:** UG1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $175,129
- **Award type:** 5
- **Project period:** 2001-09-30 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10429979

## Citation

> US National Institutes of Health, RePORTER application 10429979, Blood and Marrow Transplant Clinical Trials Network (5UG1HL069274-22). Retrieved via AI Analytics 2026-06-14 from https://api.ai-analytics.org/grant/nih/10429979. Licensed CC0.

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