Stem Cell Transplant Without Mycophenolate Mofetil for Blood Cancer

(OmitMMF Trial)

Overseen By Amy Oppenheim

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ronald Paquette

Disqualifiers: Myelofibrosis, High titer antibodies, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new approach to stem cell transplants for individuals with certain blood cancers. Researchers aim to determine if removing mycophenolate mofetil (MMF), a drug that typically suppresses the immune system, enhances the treatment's safety and effectiveness. Participants should have a blood cancer (excluding myelofibrosis) and qualify for a standard stem cell transplant from a donor. Those with prior stem cell transplants or myelofibrosis are not suitable for this trial. The study will monitor participants for up to two years post-transplant to gather results. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that omitting mycophenolate mofetil is safe for stem cell transplant patients?

Research shows that skipping mycophenolate mofetil (MMF) in stem cell transplants might be safe. Studies have examined the effects of stopping MMF and found no serious problems. Patients in these studies did not experience a significant increase in harmful effects without MMF.

Other research indicates that adjusting the combination of drugs given with MMF can still protect patients from serious issues like graft-versus-host disease (GVHD), where the new cells attack the body. These findings suggest that removing MMF from the treatment plan may not significantly increase risks.

This trial is in an early phase, testing safety but having already passed initial risk checks. This provides some confidence that the treatment could be well-tolerated. However, only specific trials can confirm its safety for everyone. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Most treatments for blood cancer following a stem cell transplant include the drug mycophenolate mofetil to prevent graft-versus-host disease (GVHD), a common complication. However, this trial is exploring the omission of mycophenolate mofetil from the treatment regimen. Researchers are excited about this approach because it could reduce the risk of side effects associated with mycophenolate mofetil, such as increased susceptibility to infections. This new method could potentially lead to a safer recovery process for patients undergoing stem cell transplants for blood cancer.

What evidence suggests that omitting mycophenolate mofetil is effective for blood cancer?

Research suggests that excluding mycophenolate mofetil (MMF) from stem cell transplant treatments might be beneficial. Some studies have shown that MMF can worsen outcomes when combined with drugs like cyclosporine. In contrast, different drug combinations without MMF have effectively managed graft-versus-host disease, a common post-transplant issue. This trial will specifically investigate the effects of omitting MMF from the treatment plan to potentially improve transplant success and reduce complications. However, further research is needed to confirm these benefits. ^{2 6 7 8 9}

Who Is on the Research Team?

Ronald Paquette, MD

Principal Investigator

Cedars-Sinai Medical Center

Are You a Good Fit for This Trial?

This trial is for patients aged 18-75 with blood cancers needing a stem cell transplant and approved by Cedars-Sinai's committee. They must be suitable for a specific chemo and radiation regimen, have not had previous transplants, and have an HLA-matched donor ready to donate stem cells.

Inclusion Criteria

I am considered fit for a specific transplant prep treatment with Fludarabine and radiation.

I am approved for a stem cell transplant for my blood cancer, not including myelofibrosis.

I have a donor who is a close genetic match for my transplant.

Exclusion Criteria

I have been diagnosed with myelofibrosis.

My donor couldn't provide enough stem cells through a blood draw.

I have high levels of antibodies against donor tissues.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Transplantation

Participants undergo allogeneic stem cell transplantation with omission of mycophenolate mofetil

Immediate

1 visit (in-patient)

Post-Transplant Monitoring

Participants are monitored for engraftment and acute GVHD up to 100 days post-transplant

100 days

Regular visits (in-person)

Follow-up

Participants are monitored for chronic GVHD, relapse, and overall survival

2 years

Periodic visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Omission of the drug mycophenolate mofetil

Trial Overview The study tests the safety of removing mycophenolate mofetil (MMF) from standard allogeneic stem cell transplant treatment in 60 patients. It aims to see if this change is feasible and beneficial over two years post-transplant without affecting graft-versus-host disease prevention.

How Is the Trial Designed?

Group I: Open Arm Experimental Treatment 1 Intervention

Omission of the drug mycophenolate mofetil

Who Is Running the Clinical Trial?

Ronald Paquette

Lead Sponsor

Trials

2

Recruited

90+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11153209/

Influence of interruption of oral mycophenolate mofetil for ... In this study, we investigated the clinical influence of oral MMF interruption on CBT outcomes at our hospital.

2. astctjournal.org astctjournal.org/article/S1083-8791(19)30330-1/fulltext

Inferior Outcomes with Cyclosporine and Mycophenolate ... Data on the comparative effectiveness of MMF compared with MTX are limited and conflicting, however. We analyzed data from the Center for International Blood ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S1083879103004804

A pilot study of tacrolimus and mycophenolate mofetil graft ... Tacrolimus (FK506)/mycophenolate mofetil (MMF) has been demonstrated to be an effective salvage therapy for steroid-resistant chronic graft-versus-host ...

4. ascopubs.org ascopubs.org/doi/abs/10.1200/JCO-25-01238

Sirolimus and Cyclosporine With Post-Transplant ... Sirolimus and Cyclosporine With Post-Transplant Cyclophosphamide or Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis in Unrelated ...

5. thelancet.com thelancet.com/journals/lanhae/article/PIIS2352-3026(24)00065-6/fulltext

a randomised, open-label, phase 3, non-inferiority trial Watchful waiting and immediate transplantation could be an alternative for fit patients with poor response or relapsed acute myeloid leukaemia ...

6. ascopubs.org ascopubs.org/doi/10.1200/JCO-25-00856

Post-Transplant Cyclophosphamide-Based Graft-Versus ... Can successful outcomes be achieved after hematopoietic stem cell transplantation (HSCT) from mismatched unrelated donors (MMUDs) using ...

7. sciencedirect.com sciencedirect.com/science/article/pii/S2666636725014472

Efficacy and Safety of Allogeneic Hematopoietic Stem Cell ... A concise review on factors influencing the hematopoietic stem cell transplantation main outcomes. Health Sci Rep, 4 (2) (2021), p. e282. View ...

8. astctjournal.org astctjournal.org/article/S1083-8791(03)00480-4/fulltext

A pilot study of tacrolimus and mycophenolate mofetil graft ... We investigated the safety and efficacy of FK506/MMF in preventing aGVHD and sparing the use of methotrexate and methylprednisolone in childhood and adolescent ...

9. nejm.org nejm.org/doi/full/10.1056/NEJMoa2503189

Graft-versus-Host Disease Prophylaxis with ... The combination of post-transplantation cyclophosphamide and a calcineurin inhibitor led to longer GVHD-free, relapse-free survival than standard prophylaxis.