Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia
Treatment Protocol: Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia Using Conditioning Regimen Without Radiation
1 other identifier
interventional
2
1 country
2
Brief Summary
Unrelated Cord Blood (UCB) transplant in children is a viable stem cell transplant modality for patients with leukemia and myelodysplasia. UCB is now considered "Standard Of Care" in cases where a suitable living bone marrow donor is not available. The survival of UCB is similar to Matched Unrelated Marrow Transplant. This study is considered "Research" since UCB is not a licensed product and requires investigational new drug (IND). THERE ARE NO SPECIFIC RESEARCH QUESTIONS IN THIS PROTOCOL. This protocol merely provides UCB as a stem cell treatment modality to pediatric patients who may require it after a conditioning regimen that excludes Total Body Irradiation.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable leukemia
Started Aug 2008
Longer than P75 for not_applicable leukemia
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 2008
CompletedFirst Submitted
Initial submission to the registry
December 3, 2013
CompletedFirst Posted
Study publicly available on registry
December 11, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2014
CompletedResults Posted
Study results publicly available
December 28, 2015
CompletedDecember 28, 2015
November 1, 2015
5.8 years
December 3, 2013
July 1, 2015
November 20, 2015
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Successful Unrelated Cord Blood (UCB) Transplants
The number of patients who received successful UCB transplants as evidenced by absolute neutrophil recovery.
2 Years
Study Arms (1)
Umbilical Cord Blood + Chemotherapy
EXPERIMENTALUmbilical Cord Blood transfusion + Chemotherapy (Fludarabine + Busulfan + Melphalan)
Interventions
Following the administration of the preparative therapy, all subjects will undergo UCB transplantation. Umbilical Cord Blood Transfusion will occur on Day 0
Fludarabine 25 mg/m2/day will be administered over 30-60 minutes intravenous infusion on Days -13 through -9 for a total of 5 doses. Fludarabine will not be dose adjusted for body weight.
Busulfan IV (Busulfex) will be administered IV every 6 hours on days -8 through -5 for a total of 16 doses. Seizure prophylaxis prior to first dose of busulfan till Day -3 will be administered.
Melphalan 45 mg/m2/day will be administered over 60 minutes intravenous infusion on Days -4 through -2 for a total of 3 doses.
Eligibility Criteria
You may qualify if:
- Patients must be up to 21 years of age
- Patients cannot receive total body irradiation (TBI) because of:
- Young age - \< 2 years at diagnosis of leukemia resulting in an age \< 4 years at transplantation (due to risk of severe growth retardation and brain damage).
- Inability to tolerate TBI because of prior radiation or organ toxicity.
- Refractory/multiply relapsed leukemia, for which a traditional TBI/cyclophosphamide regimen would unlikely lead to a successful outcome.
- Patients must have a partially human leukocyte antigen (HLA)-matched UCB unit. Unit must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the patient. The unit must deliver a pre-cryopreserved nucleated cell dose of at least 2.5 x 10\^7 per kilogram.
- Acute myelogenous leukemia (AML) at the following stages:
- High risk first complete remission (CR1), defined as:
- Having preceding myelodysplasia (MDS)
- High risk cytogenetics (High-risk cytogenetics: del (5q) -5, -7, abn (3q), t (6;9) complex karyotype (\>= 5 abnormalities)
- Requiring \> 2 cycles chemotherapy to obtain CR;
- Second or greater CR.
- First relapse with \< 25% blasts in bone marrow.
- Patients with therapy-related AML whose prior malignancy has been in remission for at least 12 months.
- Acute lymphocytic leukemia (ALL) at the following stages:
- +23 more criteria
You may not qualify if:
- Pregnant (B-positive HCG) or breastfeeding.
- Evidence of HIV infection or HIV positive serology.
- Current uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms).
- Autologous transplant \< 6 months prior to enrollment.
- Prior autologous transplant for the disease for which the UCB transplant will be performed.
- Allogeneic hematopoietic stem cell transplant \< 6 months prior to enrollment.
- Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment
- Active CNS leukemia.
- Requirement of supplemental oxygen.
- HLA-matched related donor able to donate.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Jackson Memorial Hospital
Miami, Florida, 33136, United States
University of Miami Sylvester Comprehensive Cancer Center
Miami, Florida, 33136, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
THERE ARE NO SPECIFIC RESEARCH QUESTIONS IN THIS PROTOCOL. This protocol merely provides UCB as a stem cell treatment modality to pediatric patients who may require it after a conditioning regimen that excludes Total Body Irradiation.
Results Point of Contact
- Title
- Martin Andreansky MD
- Organization
- University of Miami
Study Officials
- PRINCIPAL INVESTIGATOR
Martin Andreasky, MD, PhD
University of Miami
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Associate Professor of Clinical
Study Record Dates
First Submitted
December 3, 2013
First Posted
December 11, 2013
Study Start
August 1, 2008
Primary Completion
June 1, 2014
Study Completion
June 1, 2014
Last Updated
December 28, 2015
Results First Posted
December 28, 2015
Record last verified: 2015-11