UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep
Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Hematological Diseases Using a Non-Myeloablative Preparative Regimen
2 other identifiers
interventional
15
1 country
1
Brief Summary
This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen with modifications based on factors including diagnosis, disease status, and prior treatment. Single or double unit selected according to current University of Minnesota umbilical cord blood graft selection algorithm.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started May 2017
Longer than P75 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 24, 2016
CompletedFirst Posted
Study publicly available on registry
March 30, 2016
CompletedStudy Start
First participant enrolled
May 15, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 22, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
February 22, 2024
CompletedResults Posted
Study results publicly available
July 4, 2025
CompletedJuly 4, 2025
June 1, 2025
5.8 years
March 24, 2016
April 18, 2025
June 18, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Probability of Acute Graft Versus Host Disease (GVHD)
Simple proportions will be used to estimate the probability of grade II-IV actue GVHD.
Day 100
Secondary Outcomes (7)
Incidence of Acute GVHD
Day 100
Transplant Related Mortality
6 months post transplant
Chimerism
Day 21
Chimerism
Day 100
Chimerism
Day 180
- +2 more secondary outcomes
Study Arms (2)
No Anti-thymocyte Globulin (ATG)
EXPERIMENTALHematologic malignancy patients who have received a previous autologous transplant or ≥ 2 cycles of multi-agent chemotherapy within the 3 months previous to umbilical cord blood transplantation.
Anti-thymocyte Globulin (ATG)
EXPERIMENTALHematologic malignancy patients who have not been treated with prior autologous transplant or ≤ 1 cycle of chemotherapy in the 3 months previous to umbilical cord blood transplantation, should receive Anti-thymocyte Globulin (ATG) as part of their conditioning regimen.
Interventions
Both Arms: 30 mg/m\^2 IV over 1 hour Day -6 to Day -2
Arm 1: 50 mg/kg IV over 2 hours Day -6
Both Arms: Mycophenolate mofetil (MMF) 3 gram/day IV/PO for patients who are ≥ 40 kg divided in 2 or 3 doses. In obese patients (\>125% IBW) 15 mg/kg every 12 hours may be considered. Pediatric patient (\<40 kilograms) will receive MMF at the dose of 15 mg/kg/dose every 8 hours beginning Day -3. MMF dosing will be monitored and altered as clinically appropriate based on institutional guidelines. Patients will be eligible for MMF dosing and pharmacokinetics studies. Stop MMF at Day +30 or 7 days after engraftment, whichever day is later, if no acute graft versus host disease (GVHD). (Definition of engraftment is 1st day of 3 consecutive days of absolute neutrophil count \[ANC) ≥ 0.5 x 109 /L\]). If no donor engraftment, do not stop MMF.
Both Arms: Adult Dosing: Sirolimus will be administered starting at Day -3 with 8-12 mg oral loading dose followed by single dose 4 mg/day with a target serum concentration of 3 to 12 mg/mL by high-performance liquid chromatography (HPLC) and will be monitored per institutional guidelines. In the absence of acute GVHD sirolimus may be tapered starting at Day +100 and eliminated by Day +180 post-transplantation. Pediatric Dosing: Sirolimus will be administered starting on Day -3 with an oral loading dose of 10 mg followed by maintenance dosing of 2.5 mg/m\^2/day (Maximum total daily dose of 4mg) as per institutional guidelines. Target serum concentration goals are 3 to 12 mg/mL by high-performance liquid chromatography (HPLC) and will be monitored per institutional guidelines.
Both Arms: 200 cGy on Day -1
Both Arms: Day 0
Arm 2: 15 mg/kg IV every 12 hours Day -6 to Day -4
Eligibility Criteria
You may qualify if:
- Age, Performance Status, and Graft Criteria
- \<70 years of age with no matched 5/6 or 6/6 sibling donor - patients ≥ 70 and ≤ 75 years of age may be eligible if they have a Co-Morbidity score ≤ 2 (http://www.qxmd.com/calculate-online/hematology/hct-ci)
- Karnofsky score ≥ 70% (≥ 16 years) or Lansky score ≥ 50 (\< 16 years)
- UCB graft selected according to current University of Minnesota umbilical cord blood graft selection algorithm
- Eligible Diseases All diseases listed below are advanced hematologic malignancies not curable by conventional chemotherapy. Responses to conventional treatment range from zero to 30% but are typically short lived.
- Acute Leukemias: Must be in remission by morphology (\<5% blasts). Note cytogenetic relapse or persistent disease without morphologic relapse is acceptable. Also a small percentage of blasts that is equivocal between marrow regeneration vs. early relapse are acceptable provided there are no associated cytogenetic markers consistent with relapse.
- Acute Myeloid Leukemia (AML) and related precursor neoplasms: 2nd or greater complete remission (CR); first complete remission (CR1) in patients \> 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk. Favorable risk AML is defined as having one of the following:
- t(8,21) without cKIT mutation
- inv(16) or t(16;16) without cKIT mutation
- Normal karyotype with mutated NPM1 and wild type FLT-ITD
- Normal karyotype with double mutated CEBPA
- Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation
- Acute lymphoblastic leukemia (ALL)/lymphoma: second or greater CR; CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL. High risk ALL is defined as having one of the following:
- Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1
- years of age or older at diagnosis
- +31 more criteria
You may not qualify if:
- Pregnant or breast feeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy.
- Untreated active infection
- Active HIV infection or known HIV positive serology
- Less than 3 months since prior myeloablative transplant
- CML in blast crisis
- Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressing on salvage therapy.
- Active central nervous system malignancy
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, 55455, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Margaret MacMillan
- Organization
- University of Minnesota, Masonic Cancer Center
Study Officials
- PRINCIPAL INVESTIGATOR
Margaret MacMillan, MD, MSC
University of Minnesota
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 24, 2016
First Posted
March 30, 2016
Study Start
May 15, 2017
Primary Completion
February 22, 2023
Study Completion
February 22, 2024
Last Updated
July 4, 2025
Results First Posted
July 4, 2025
Record last verified: 2025-06