Mobilized Peripheral Blood Stem Cell graft with Tacrolimus/Mycophenolate Mofetil/Post-Transplant Cyclophosphamide for Myelodysplasia

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Myelodysplasia+8 More
Mobilized Peripheral Blood Stem Cell graft with Tacrolimus/Mycophenolate Mofetil/Post-Transplant Cyclophosphamide - Procedure
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This trial is comparing two ways to prevent GVHD, a complication of stem cell transplants. One group will receive tacrolimus/methotrexate and the other group will receive post-transplant cyclophosphamide/tacrolimus/mycophenolate mofetil. The trial will also test the hypothesis that the engraftment stool microbiome diversity predicts one-year non-relapse mortality.

Eligible Conditions
  • Myelodysplasia
  • leukemia
  • Lymphoma

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 15 Secondary · Reporting Duration: 1 year

1 year
Chronic GVHD
Disease Relapse or Progression
Disease-free Survival
GVHD-free, Relapse-free survival (GRFS) probability
Immunosuppression-free Survival
Infections
Overall Survival
Post-Transplant Lymphoproliferative Disease (PTLD)
Toxicity grade over time
Month 24
Diversity and Composition of the Gut Microbiome
Diversity of Immune Repertoire
Year 1
Patient-Reported Outcomes (PRO)
Transplant-related Mortality
Year 1
Graft-vs-Host Disease
Days 28 and 100
Donor Cell Engraftment
Hematologic Recovery

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

2 Treatment Groups

Tacrolimus/Methotrexate
1 of 2
Tacrolimus/MMF/PTCY
1 of 2
Active Control
Experimental Treatment

428 Total Participants · 2 Treatment Groups

Primary Treatment: Mobilized Peripheral Blood Stem Cell graft with Tacrolimus/Mycophenolate Mofetil/Post-Transplant Cyclophosphamide · No Placebo Group · Phase 3

Tacrolimus/MMF/PTCYExperimental Group · 4 Interventions: Tacrolimus, Mycophenolate Mofetil, Mobilized Peripheral Blood Stem Cell graft with Tacrolimus/Mycophenolate Mofetil/Post-Transplant Cyclophosphamide, Cyclophosphamide · Intervention Types: Drug, Drug, Procedure, Drug
Tacrolimus/MethotrexateActiveComparator Group · 3 Interventions: Tacrolimus, Methotrexate, Mobilized Peripheral Blood Stem Cell graft with Tacrolimus/Methotrexate · Intervention Types: Drug, Drug, Procedure
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tacrolimus
FDA approved
Mycophenolate mofetil
FDA approved
Cyclophosphamide
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year

Who is running the clinical trial?

National Marrow Donor ProgramOTHER
59 Previous Clinical Trials
182,135 Total Patients Enrolled
1 Trials studying Myelodysplasia
340 Patients Enrolled for Myelodysplasia
National Cancer Institute (NCI)NIH
12,990 Previous Clinical Trials
41,298,460 Total Patients Enrolled
3 Trials studying Myelodysplasia
2,125 Patients Enrolled for Myelodysplasia
Medical College of WisconsinLead Sponsor
554 Previous Clinical Trials
1,151,744 Total Patients Enrolled
Blood and Marrow Transplant Clinical Trials NetworkNETWORK
49 Previous Clinical Trials
13,833 Total Patients Enrolled
2 Trials studying Myelodysplasia
625 Patients Enrolled for Myelodysplasia
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,576 Previous Clinical Trials
46,968,153 Total Patients Enrolled
2 Trials studying Myelodysplasia
625 Patients Enrolled for Myelodysplasia
Mary Horowitz, MD, MSStudy DirectorCenter for International Blood and Marrow Transplant Research
12 Previous Clinical Trials
3,502 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You must be at least 18 years old to join Segment A.
This text refers to patients who have leukemia and have less than 5% blasts in their bone marrow.
had a significantly better outcome than those with chemoresistant disease Patients who had chemosensitive disease at the time of their transplantation had a significantly better outcome than those who had chemoresistant disease.
are eligible for allogeneic hematopoietic cell transplant (HCT) from a matched sibling donor Patients with myelodysplasia/chronic myelomonocytic leukemia who have no circulating blasts and less than 10% blasts in their bone marrow are eligible for allogeneic hematopoietic cell transplant from a matched sibling donor.
had a 5-year overall survival of 82% The five-year survival rate for patients with lymphoma who had chemosensitive disease at the time of transplantation was eighty-two percent.
A sibling donor must be a 6/6 match for Human Leukocyte Antigen-A (HLA)-A and -B at intermediate (or higher) resolution, and -DRB1 at high resolution using DNA-based typing, and must be willing to donate peripheral blood stem cells and meet institutional criteria for donation.
An unrelated donor must have a 7/8 or 8/8 HLA-A, -B, -C, and -DRB1 match at high resolution using DNA-based typing
is 30 to 50 ml/min/1.73 m2 The estimated creatinine clearance acceptable per institutional guidelines is 30 to 50 ml/min/1.73 m2.
A planned reduced intensity conditioning regimen is an acceptable treatment option for people with AML
The patient's cardiac function is adequate, with a left ventricular ejection fraction of at least 45%.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 31st, 2021

Last Reviewed: October 16th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.