# Blood and Marrow Transplant Clinical Trials Network (BMT CTN) - Core Clinical Centers - UG1

> **NIH NIH UG1** · H. LEE MOFFITT CANCER CTR & RES INST · 2024 · $214,179

## Abstract

Project Summary:
The goal of the Blood and Marrow Transplant Clinical Trial Network (BMT CTN) is to generate efficacy and
safety data from multicenter Phase II and III trials, and advance the science and technology of immune and
hematopoietic cellular therapies for the cure of blood disorders. The Moffitt Consortium brings scientific
leadership, expertise in research conduct, and robust clinical volumes to support the mission of the BMT CTN.
We also submit a project to specifically address major knowledge gaps in the domain of CAR-T therapy,
namely the optimal lymphodepleting chemotherapy approach. We propose a national randomized phase II trial
to identify an alternative lymphodepleting chemotherapy (LD-chemo) approach that optimizes efficacy and
safety in the setting of CD19 CAR-T therapy for large B cell lymphoma (utilizing two approved, commercially
available products, axicabtagene ciloleucel (axi-cel) and lisocabtagene maraleucel (liso-cel). The study arms
include 1) fludarabine/cyclophosphamide (Flu/Cy) with fludarabine dosing optimized using mathematical
modeling of fludarabine exposure, 2) Bendamustine (Benda), and 3) Cladribine/Cy (Clad/Cy). Each of these
approaches has been studied in single center series and have evidence in support of improved outcomes. Our
proposed trial will for the first time prospectively test these regimens to determine which outperforms traditional
Flu/Cy for efficacy (12 month progression-free survival) and toxicity (composite severe toxicity endpoint
inclusive of grade 3 or higher cytokine release syndrome (CRS) and immune cell therapy associated
neurologic toxicity (ICANS), day 30 severe cytopenias, and severe infection). In parallel, we will interrogate the
biologic differences in immune cells and homeostatic cytokines in each study arm and correlate these findings
with clinical outcomes. This highly innovative trial has major potential to determine which of these LD-chemo
approaches optimizes outcomes, and will inform selection of an approach to be tested in a subsequent
randomized phase III trial against conventional standard of care Flu/Cy. The findings will have major impact on
clinical practice, the current state of the science, and potential to directly improve patient outcomes.

## Key facts

- **NIH application ID:** 10939818
- **Project number:** 2UG1HL108987-14
- **Recipient organization:** H. LEE MOFFITT CANCER CTR & RES INST
- **Principal Investigator:** Joseph A Pidala
- **Activity code:** UG1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $214,179
- **Award type:** 2
- **Project period:** 2011-08-08 → 2031-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10939818

## Citation

> US National Institutes of Health, RePORTER application 10939818, Blood and Marrow Transplant Clinical Trials Network (BMT CTN) - Core Clinical Centers - UG1 (2UG1HL108987-14). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10939818. Licensed CC0.

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