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

Trial Summary

What is the purpose of this trial?

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.

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 data supports the effectiveness of omitting the drug mycophenolate mofetil in stem cell transplants for blood cancer?

The research does not provide direct evidence about omitting mycophenolate mofetil in stem cell transplants for blood cancer, but it highlights that mycophenolate mofetil is a strong immunosuppressant with potential side effects like infections, which might be a reason to explore its omission in certain treatments. ^{1 2 3 4 5}

Is it safe to omit mycophenolate mofetil in stem cell transplants for blood cancer?

The safety of omitting mycophenolate mofetil in stem cell transplants for blood cancer isn't directly addressed in the available research. However, mycophenolate mofetil itself is known to cause side effects like gastrointestinal issues and increased risk of infections, so omitting it might reduce these risks, but more specific studies are needed to confirm safety in this context. ^{6 7 8 9 10}

How does omitting the drug mycophenolate mofetil in stem cell transplants for blood cancer differ from other treatments?

This treatment is unique because it omits mycophenolate mofetil, a common immunosuppressive drug used to prevent organ rejection by inhibiting immune cell proliferation. By not using this drug, the treatment may reduce side effects like gastrointestinal issues and infections, which are associated with mycophenolate mofetil. ^{3 6 7 8 11}

Research Team

Ronald Paquette, MD

Principal Investigator

Cedars-Sinai Medical Center

Eligibility Criteria

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

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)

Treatment Details

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.

Participant Groups

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+

References

1. Denmark pubmed.ncbi.nlm.nih.gov

Prevalence of mycophenolate mofetil discontinuation and subsequent outcomes in pediatric kidney transplant recipients: A PNRC study. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Does the evidence support the use of mycophenolate mofetil therapeutic drug monitoring in clinical practice? A systematic review. [2018]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Mycophenolate mofetil, Cellcept, a new immunosuppressive drug with great potential in internal medicine. [2019]

4. Czech Republic pubmed.ncbi.nlm.nih.gov

[Mycophenolate mofetil, a possible new immunosuppresant]. [2016]

5. Australia pubmed.ncbi.nlm.nih.gov

Safe conversion of mycophenolate mofetil to azathioprine in kidney transplant recipients with sirolimus-based immunosuppression. [2016]

6. New Zealand pubmed.ncbi.nlm.nih.gov

Preliminary risk-benefit assessment of mycophenolate mofetil in transplant rejection. [2018]

7. Norway pubmed.ncbi.nlm.nih.gov

[Mycophenolate mofetil--a new immunosuppressive agent]. [2016]

8. United States pubmed.ncbi.nlm.nih.gov

The use of mycophenolate mofetil (RS-61443) in human heart transplant recipients. [2019]

9. United States pubmed.ncbi.nlm.nih.gov

Morbidity rates with reduced mycophenolate mofetil dosage: a single-center experience. [2016]

10. New Zealand pubmed.ncbi.nlm.nih.gov

Adverse gastrointestinal effects of mycophenolate mofetil: aetiology, incidence and management. [2018]

11. New Zealand pubmed.ncbi.nlm.nih.gov

Mycophenolate mofetil: a review of its use in the management of solid organ transplantation. [2018]