Back to resultsFludarabine 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 (OmitMMF)
Primary Purpose
Hematologic Neoplasms
Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Omission of the drug mycophenolate mofetil
Sponsored by
About this trial
This is an interventional treatment trial for Hematologic Neoplasms focused on measuring allogeneic stem cell transplant
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 and has been approved by the Cedars-Sinai Blood and Marrow Transplant Selection Committee (separate clinical consent for the standard of care transplant will be obtained at the time of research consent).
- 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
Sites / Locations
- Cedars-Sinai Medical CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Open Arm
Arm Description
Omission of the drug mycophenolate mofetil
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Time to neutrophil and platelet engraftment
Days to neutrophil and platelet engraftment since transplant.
Rate of severe acute GVHD by day +100.
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
Rate of treatment-related mortality
Rate of severe chronic GVHD at 1 year
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
Relapse rate at 1 and 2 years
Overall Survival (OS)
Overall survival at 1 and 2 years
Graft Versus Host Disease (GVHD)-free, Relapse Free Survival
Rate of GVHD-free, Relapse Free Survival
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05256537
Brief Title
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
Acronym
OmitMMF
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
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 26, 2022 (Actual)
Primary Completion Date
September 30, 2023 (Anticipated)
Study Completion Date
September 30, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Ronald Paquette
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hematologic Neoplasms
Keywords
allogeneic stem cell transplant
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Open-label safety trial
Masking
None (Open Label)
Allocation
N/A
Enrollment
60 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Open Arm
Arm Type
Experimental
Arm Description
Omission of the drug mycophenolate mofetil
Intervention Type
Other
Intervention Name(s)
Omission of the drug mycophenolate mofetil
Intervention Description
Elimination of the immunosuppressive drug mycophenolate mofetil (MMF) from the post-transplant regimen.
Primary Outcome Measure Information:
Title
Engraftment failure
Description
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.
Time Frame
60 days post-transplant
Secondary Outcome Measure Information:
Title
Time to neutrophil and platelet engraftment
Description
Days to neutrophil and platelet engraftment since transplant.
Time Frame
60 days post-transplant
Title
Rate of severe acute GVHD by day +100.
Description
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.
Time Frame
100 days post-transplant
Title
Treatment-related mortality
Description
Rate of treatment-related mortality
Time Frame
5 days post- transplant to 2-years
Title
Rate of severe chronic GVHD at 1 year
Description
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.
Time Frame
1-year post-transplant
Title
Relapse
Description
Relapse rate at 1 and 2 years
Time Frame
1 year post-transplant and 2 years post-transplant
Title
Overall Survival (OS)
Description
Overall survival at 1 and 2 years
Time Frame
1-year post-transplant and 2-years post-transplant
Title
Graft Versus Host Disease (GVHD)-free, Relapse Free Survival
Description
Rate of GVHD-free, Relapse Free Survival
Time Frame
1-year post- transplant and 2-years post-transplant
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Amy Oppenheim
Phone
310-423-3713
Email
Amy.Oppenheim@cshs.org
First Name & Middle Initial & Last Name or Official Title & Degree
Ronald Paquette, MD
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ronald Paquette, MD
Organizational Affiliation
Cedars-Sinai Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cedars-Sinai Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Amy Oppenheim
Phone
310-423-3713
Email
Amy.Oppenheim@cshs.org
First Name & Middle Initial & Last Name & Degree
John Chute, MD
First Name & Middle Initial & Last Name & Degree
Behrooz Hakimian, MD
First Name & Middle Initial & Last Name & Degree
Justin Darrah, MD
First Name & Middle Initial & Last Name & Degree
Akil Merchant, MD
First Name & Middle Initial & Last Name & Degree
Noah Merin, MD
First Name & Middle Initial & Last Name & Degree
Josh Sasine, MD
First Name & Middle Initial & Last Name & Degree
Robert Vescio, MD
12. IPD Sharing Statement
Learn more about this trial
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
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