Fludarabine and Total Body Irradiation 800 Centigray (cGy) or 1125 cGy For Allogeneic Stem Cell Transplant Using Graft Versus Host Disease Prophylaxis With Post-Transplant Cyclophosphamide and Tacrolimus, Without Mycophenolate Mofetil

NCT05256537 | Active Not Recruiting Phase 2 DMC

• 1 other identifier: IIT2021-11-PAQUETTE-OmitMMF
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

This is a pilot study to evaluate the feasibility, safety and potential benefits of removing one immune suppressive drug called mycophenolate mofetil (MMF) from the standard allogenic stem cell transplant treatment protocol. MMF will be omitted from the transplant regimen in 60 eligible patients with hematologic malignancies. Participants will be followed for up to 2 years post standard of care transplant at Cedars-Sinai.

Conditions

Hematologic Neoplasms

Keywords

allogeneic stem cell transplant

Outcome Measures

Primary Outcomes (1)

• Engraftment failure

  Evaluate the feasibility of eliminating the drug Mycophenolate Mofetil (MMF) from the transplant regimens as determined by an acceptable rate of engraftment failure (\<10%). Engraftment failure is when the blood-forming cells received on transplant day do not start to grow and make healthy blood cells.

  60 days post-transplant

Secondary Outcomes (7)

• Time to neutrophil and platelet engraftment

  60 days post-transplant

• Rate of severe acute GVHD by day +100.

  100 days post-transplant

• Treatment-related mortality

  5 days post- transplant to 2-years

• Rate of severe chronic GVHD at 1 year

  1-year post-transplant

• Relapse

  1 year post-transplant and 2 years post-transplant

Study Arms (1)

Open Arm

EXPERIMENTAL

Omission of the drug mycophenolate mofetil

Other: Omission of the drug mycophenolate mofetil

Interventions

Omission of the drug mycophenolate mofetil OTHER

Elimination of the immunosuppressive drug mycophenolate mofetil (MMF) from the post-transplant regimen.

Open Arm

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient age 18-75 years
• Patient has a related donor who is at least Human Leukocyte Antigen (HLA) haploidentical, or an unrelated donor who is a most a single HLA antigen mismatch.
• Patient signs the Informed Consent Form for the study
• Patient has a hematologic malignancy other than myelofibrosis and meets standard criteria for allogeneic stem cell transplant.
• Patient is deemed suitable to receive Fludarabine and Total Body Irradiation (Flu/TBI) 1125 or Flu/TBI 800 conditioning regimen as standard of care transplant
• Donor is willing to donate peripheral blood stem cells

You may not qualify if:

• Patient has a diagnosis of myelofibrosis
• Patient has high titer antibodies against one or more donor HLA antigens
• Patient has undergone prior autologous or allogeneic stem cell transplant.
• Inability to collect sufficient peripheral blood stem cells from the donor

Sponsors & Collaborators

• Ronald Paquette: lead

Study Sites (1)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Ronald Paquette, MD

  Cedars-Sinai Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Medical Director, Blood and Marrow Transplant Program

Model Details: Open-label safety trial

Study Record Dates

• First Submitted: January 27, 2022
• First Posted: February 25, 2022
• Study Start: April 26, 2022
• Primary Completion: September 20, 2024
• Study Completion: March 7, 2026
• Last Updated: December 8, 2025

Record last verified: 2025-12

Locations

US (1)