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Alkylating agents

Chemotherapy + Stem Cell Transplant for Blood Cancers

Phase 2

Waitlist Available

Led By Uday R Popat

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Left ventricular ejection fraction >= 50%

Adequate pulmonary function with forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC) and diffusion capacity of the lung for carbon monoxide (DLCO) >= 50% of expected corrected for hemoglobin and/or volume; children unable to perform pulmonary function tests (e.g., less than 7 years old) pulse oximetry of >= 92% on room air

Must not have

Patients who received inotuzumab and/or gemtuzumab in the past

Human immunodeficiency virus (HIV) seropositivity

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trialstudies the side effects of drugs used to stop the growth of cancer cells before & after a stem cell transplant.

Who is the study for?

This trial is for patients with high-risk blood cancers like various leukemias, lymphomas, and myeloma. They should have a poor prognosis without transplant therapy and can be in remission or relapsed. Participants need functioning major organs, no active hepatitis B/C or HIV, not pregnant nor breastfeeding, willing to use contraception if applicable, and must have a suitable donor for stem cell transplant.

What is being tested?

The study tests busulfan and fludarabine phosphate chemotherapy followed by donor stem cell transplant with post-transplant cyclophosphamide treatment. It aims to see how well these drugs work together to stop cancer growth by killing cells or preventing their spread while reducing the risk of graft-versus-host disease.

What are the potential side effects?

Potential side effects include damage to bone marrow (affecting blood cell production), immune system reactions against normal body cells (graft-versus-host disease), organ inflammation from the immune response, increased infection risk due to weakened immunity, nausea, hair loss from chemotherapy agents used.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My heart pumps blood well.

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My lung function tests are within normal limits, or if I'm a child under 7, my oxygen level is 92% or higher on room air.

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My kidneys are functioning well enough to filter waste.

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I have a high-risk blood cancer with a poor outlook without transplant.

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I have a closely matched or half-matched family member or unrelated donor for my treatment.

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I am mostly active and can care for myself.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have previously been treated with inotuzumab or gemtuzumab.

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I am HIV positive.

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I have had a transplant from a donor.

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I have active hepatitis B or C.

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I have had heart disease related to my arteries.

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I do not have any infections that are not responding to treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Non-relapse mortality rate

Secondary study objectives

Graft versus host disease-free survival/relapse free survival

Bone Transplantation

Incidence of grade 3 and 4 adverse events

+5 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

6Treatment groups

Experimental Treatment

Group I: Group VI (matched or haploidentical transplant, chemotherapy)Experimental Treatment10 Interventions

Patients receiving fully matched or haploidentical donor transplant receive busulfan IV over 3 hours on days -20, -13, and -6 to -3, a lower dose of thiotepa IV over 4 hours on day -7, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months and mycophenolate mofetil PO TID.

Group II: Group V (haploidentical donor transplant, chemotherapy)Experimental Treatment10 Interventions

Patients receive busulfan IV over 3 hours on days -20, -13, and -6 to -3, a lower dose of thiotepa IV over 4 hours on day -7, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months and mycophenolate mofetil PO TID.

Group III: Group IV (matched donor transplant, chemotherapy)Experimental Treatment8 Interventions

Patients receiving haploidentical related donor transplant, diagnosis of myelofibrosis, \> 60 years old, or patients with comorbidity scores \> 3 will go in Group 3 or 4. If patients with comorbidity score \>3, then the principal investigator is the final arbiter of eligibility for comorbidity score \> 3. Busulfan is administered at the dose calculated to achieve a total (including first two doses delivered on day -20 and day -13) system exposure of 20,000 +/- 12% uMol-min based on the pharmacokinetic studies.

Group IV: Group III (haploidentical donor transplant, chemotherapy)Experimental Treatment10 Interventions

Patients receiving haploidentical related donor transplant, diagnosis of myelofibrosis, \> 60 years old, or patients with comorbidity scores \> 3 will go in Group 3 or 4. If patients with comorbidity score \> 3, then the principal investigator is the final arbiter of eligibility for comorbidity score \> 3. Busulfan is administered at the dose calculated to achieve a total (including first two doses delivered on day -20 and day -13) system exposure of 20,000 +/- 12% uMol-min based on the pharmacokinetic studies.

Group V: Group II (matched donor transplant, chemotherapy)Experimental Treatment8 Interventions

Patients receive busulfan IV over 3 hours on days -13, -12, and -6 to -3, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months.

Group VI: Group I (haploidentical donor transplant, chemotherapy)Experimental Treatment10 Interventions

Patients receive busulfan IV over 3 hours on days -13, -12, and -6 to -3, thiotepa IV over 4 hours on day -7, fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo stem cell transplantation IV on day 0. Patients then receive cyclophosphamide IV over 3 hours on days 3 and 4. Beginning on day 5, patients receive tacrolimus IV continuously or PO BID for up to 3 months and mycophenolate mofetil PO TID.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Busulfan

2008

Completed Phase 4

~1710

Tacrolimus

2019

Completed Phase 4

~5510