Hematologic Neoplasms
Conditions
Keywords
allogeneic stem cell transplant
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.
Interventions
OTHEROmission of the drug mycophenolate mofetil
Elimination of the immunosuppressive drug mycophenolate mofetil (MMF) from the post-transplant regimen.
Sponsors
Ronald Paquette
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-label safety trial
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Engraftment failure | 60 days post-transplant | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to neutrophil and platelet engraftment | 60 days post-transplant | Days to neutrophil and platelet engraftment since transplant. |
| Rate of severe acute GVHD by day +100. | 100 days post-transplant | Proportion of patients with severe acute GVHD at day 100 post-transplantation. * Acute GVHD is when the donated stem cells attack the body within the first 100 days post-transplant. * The modified Keystone criteria will be used for acute GVHD staging and grading. |
| Treatment-related mortality | 5 days post- transplant to 2-years | Rate of treatment-related mortality |
| Rate of severe chronic GVHD at 1 year | 1-year post-transplant | Proportion of patients with severe chronic GVHD at day 365 post-transplantation. -Chronic GVHD is when the donated stem cells attack the body after 100 days post-transplant. Chronic GVHD is defined by NIH Consensus Criteria for chronic GVHD. |
| Relapse | 1 year post-transplant and 2 years post-transplant | Relapse rate at 1 and 2 years |
| Overall Survival (OS) | 1-year post-transplant and 2-years post-transplant | Overall survival at 1 and 2 years |
| Graft Versus Host Disease (GVHD)-free, Relapse Free Survival | 1-year post- transplant and 2-years post-transplant | Rate of GVHD-free, Relapse Free Survival |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORRonald Paquette, MD
Cedars-Sinai Medical Center
Outcome results
None listed