Hematologic NeoplasmsPhase 2OmitMMFNCT05256537
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
A Phase 2 study testing Omission of the drug mycophenolate mofetil in people with Hematologic Neoplasms, run by Ronald Paquette at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Hematologic Neoplasms
- What is being tested?
- Omission of the drug mycophenolate mofetil
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Apr 2022to 7 Jan 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Cedars-Sinai Medical Center
Los Angeles, California, 90048
About this study
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
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
- Being tested
- Omission of the drug mycophenolate mofetil
- Keywords
- allogeneic stem cell transplant
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Omission of the drug mycophenolate mofetilOther
Elimination of the immunosuppressive drug mycophenolate mofetil (MMF) from the post-transplant regimen.
| Study group | Role | What they receive |
|---|---|---|
| Open Arm | Experimental | Omission of the drug mycophenolate mofetil Receives: Other: Omission of the drug mycophenolate mofetil |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 60 days post-transplant
Secondary outcomes (7)
Time to neutrophil and platelet engraftment
Days to neutrophil and platelet engraftment since transplant.
Measured: 60 days post-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.
Measured: 100 days post-transplant
Treatment-related mortality
Rate of treatment-related mortality
Measured: 5 days post- transplant to 2-years
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.
Measured: 1-year post-transplant
Relapse
Relapse rate at 1 and 2 years
Measured: 1 year post-transplant and 2 years post-transplant
Overall Survival (OS)
Overall survival at 1 and 2 years
Measured: 1-year post-transplant and 2-years post-transplant
Graft Versus Host Disease (GVHD)-free, Relapse Free Survival
Rate of GVHD-free, Relapse Free Survival
Measured: 1-year post- transplant and 2-years post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2022
- Study start26 Apr 2022
- Primary completion20 Sep 2024
- Study completion7 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic NeoplasmsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label safety trial
- Masking
- None (open label)
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ronald Paquette
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ronald Paquette, Cedars-Sinai Medical Center
- Investigators
- Ronald Paquette, MD, Principal investigator, Cedars-Sinai Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05256537
- Sponsor's protocol ID
- IIT2021-11-PAQUETTE-OmitMMF
- Acronym
- OmitMMF
- Status last verified
- Aug 2026
- First submitted
- 27 Jan 2022
- Last updated
- 5 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05256537
- Acronym / sponsor ID
- OmitMMF / IIT2021-11-PAQUETTE-OmitMMF
- Conditions
- Hematologic Neoplasms
- Keywords
- allogeneic stem cell transplant
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Ronald PaquetteUniversity, hospital or other
- Study start
- 26 Apr 2022 (actual)
- Primary completion
- 20 Sep 2024 (actual)
- Study completion
- 7 Jan 2027 (estimated)
- First posted
- 25 Feb 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label safety trial
| Arm | Type | Receives |
|---|---|---|
| Open Arm | Experimental | Other: Omission of the drug mycophenolate mofetilOmission of the drug mycophenolate mofetil |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Omission of the drug mycophenolate mofetilElimination of the immunosuppressive drug mycophenolate mofetil (MMF) from the post-transplant regimen. | Other | Open Arm |
Primary outcome
Engraftment failure
Time frame: 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.
7 secondary outcomes
Time to neutrophil and platelet engraftment
Time frame: 60 days post-transplant
Rate of severe acute GVHD by day +100.
Time frame: 100 days post-transplant
Treatment-related mortality
Time frame: 5 days post- transplant to 2-years
Rate of severe chronic GVHD at 1 year
Time frame: 1-year post-transplant
Relapse
Time frame: 1 year post-transplant and 2 years post-transplant
Overall Survival (OS)
Time frame: 1-year post-transplant and 2-years post-transplant
Graft Versus Host Disease (GVHD)-free, Relapse Free Survival
Time frame: 1-year post- transplant and 2-years post-transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ronald Paquette, MD principal investigator
Cedars-Sinai Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Omission of the drug mycophenolate mofetil in people with Hematologic Neoplasms, run by Ronald Paquette at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 5 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Engraftment failure (time frame: 60 days post-transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2026.