Clinical trial NCT05256537: 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
Official title: IIT2021-11-PAQUETTE-OmitMMF: Fludarabine and Total Body Irradiation 800 cGy or 1125 cGy For Allogeneic Stem Cell Transplant Using Graft Versus Host Disease Prophylaxis With Post-Transplant Cyclophosphamide and Tacrolimus, Without Mycophenolate Mofetil
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Hematologic Neoplasms
Keywords: allogeneic stem cell transplant
Lead sponsor: Cedars-Sinai Medical Center
Sponsor class: OTHER
First posted: 2022-02-25
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-label safety trial
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Engraftment failure
timeFrame: 60 days post-transplant
Secondary outcomes:
measure: Time to neutrophil and platelet engraftment
timeFrame: 60 days post-transplant
measure: Rate of severe acute GVHD by day +100.
timeFrame: 100 days post-transplant
measure: Treatment-related mortality
timeFrame: 5 days post- transplant to 2-years
measure: Rate of severe chronic GVHD at 1 year
timeFrame: 1-year post-transplant
measure: Relapse
timeFrame: 1 year post-transplant and 2 years post-transplant
measure: Overall Survival (OS)
timeFrame: 1-year post-transplant and 2-years post-transplant
measure: Graft Versus Host Disease (GVHD)-free, Relapse Free Survival
timeFrame: 1-year post- transplant and 2-years post-transplant
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
Study officials:
affiliation: Cedars-Sinai Medical Center
name: Ronald Paquette, MD
role: PRINCIPAL_INVESTIGATOR
References: