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Alkylating agents

Stem Cell Transplant for Infant Leukemia

Phase 2
Waitlist Available
Led By Christen Ebens, MD
Research Sponsored by Masonic Cancer Center, University of Minnesota
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
High risk cytogenetics t(4;11) or other MLL rearrangements; chromosome 5, 7, or 19 abnormalities; complex karyotype (>5 distinct changes); ≥ 2 cycles to obtain complete response (CR); CR2 or higher; Preceding myelodysplastic syndrome (MDS); All patients must be in CR or early relapse (i.e., <15% blasts in BM).
Acute lymphocytic leukemia: high risk CR1 as evidenced by: High-risk cytogenetic: t(4;11) or other MLL rearrangements; hypodiploid; t(9;22); >1 cycle to obtain CR; CR2 or higher; All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
Must not have
History of HIV infection or known positive serology
Evidence of active extramedullary disease (including central nervous system leukemia).
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 1 and 2 after transplant
Awards & highlights
No Placebo-Only Group

Summary

This trial is studying how well combination chemotherapy followed by a donor umbilical cord blood transplant works in treating infants with high-risk acute leukemia or myelodysplastic syndromes.

Who is the study for?
This trial is for infants under 3 years old with high-risk acute leukemia or myelodysplastic syndromes. They must be in complete remission or early relapse, have a matched donor for stem cell transplant, and good organ function. Infants can't join if they have an active infection, HIV, extramedullary disease, or had a myeloablative transplant within the last 6 months.
What is being tested?
The study tests how well chemotherapy (busulfan, fludarabine, melphalan) followed by umbilical cord blood stem cell transplant works in treating infant leukemia. It also looks at preventing immune reactions against normal cells using cyclosporine and mycophenolate mofetil after the transplant.
What are the potential side effects?
Possible side effects include reactions to chemotherapy like nausea and hair loss; complications from stem cell transplantation such as infections; and immune system's possible attack on the body's own cells despite preventive medication.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My leukemia has high-risk features and is either in remission or early relapse.
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My leukemia is high risk, in remission, and my bone marrow shows less than 5% blasts.
Select...
My liver tests are within 5 times the normal range.
Select...
My kidney function is good, with a filtration rate over 60.
Select...
My blood disorder is considered high risk but with less than 10% immature blood cells.
Select...
My transplant match is almost perfect and the cell dose is high enough.
Select...
I can do most activities and my organs work well.
Select...
My transplant match is perfect and the cell dose is high enough.
Select...
My heart pumps well, with an ejection fraction over 45%.
Select...
My treatment requires a specific match and cell dose.
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My acute myeloid leukemia is considered high risk.
Select...
My cancer remains or is increasing despite having standard chemotherapy.
Select...
I was diagnosed with a blood cancer before I turned 4 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have a history of HIV or tested positive for it.
Select...
My leukemia has spread outside the bone marrow.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 1 and 2 years after transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1 and 2 years after transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Incidence of Engraftment
Secondary study objectives
Developmental Outcomes
Disease-free survival
Incidence of acute graft-versus-host disease (GVHD) grade II-IV and grade III-IV
+5 more

Side effects data

From 2013 Phase 2 & 3 trial • 7 Patients • NCT00775931
100%
Acute Sinusitis
100%
Atelectasis
100%
Fungal Infection, Respiratory
100%
Pericardial Effusion
100%
Bacterial Infection, Respiratory
100%
Bacterial Infection, Feces
100%
Blood Clot in Bladder Wall
100%
Fungal Infection, Feces
100%
Requires Hemodialysis, NOS
100%
Hyperechogenic Renomegaly with Ascites
100%
Requires Intubation, NOS
100%
Large Splenic Infarct
100%
Low Lung Volumes
100%
Pneumonia
100%
Pulmonary Hemorrhage
100%
Small Bowel Ascites
100%
Viral Infection, Blood
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cord Blood Transplant Conditioning
Marrow Graft Transplant Conditioning

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: Single Unit UCB TransplantationExperimental Treatment7 Interventions
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
Group II: Double Unit UCB TransplantationExperimental Treatment7 Interventions
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
fludarabine phosphate
2000
Completed Phase 3
~2660
cyclosporine
2000
Completed Phase 3
~1290
melphalan
1994
Completed Phase 3
~3530
mycophenolate mofetil
2000
Completed Phase 3
~1740
umbilical cord blood transplantation
2008
Completed Phase 3
~1130
filgrastim
1997
Completed Phase 3
~7260
busulfan
1999
Completed Phase 3
~3630

Find a Location

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor
279 Previous Clinical Trials
15,548 Total Patients Enrolled
74 Trials studying Leukemia
3,299 Patients Enrolled for Leukemia
Christen Ebens, MDPrincipal InvestigatorMasonic Cancer Center, University of Minnesota
1 Previous Clinical Trials
30 Total Patients Enrolled

Media Library

Busulfan (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT00357565 — Phase 2
Leukemia Research Study Groups: Double Unit UCB Transplantation, Single Unit UCB Transplantation
Leukemia Clinical Trial 2023: Busulfan Highlights & Side Effects. Trial Name: NCT00357565 — Phase 2
Busulfan (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT00357565 — Phase 2
~2 spots leftby Nov 2025
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