Umbilical Cord Blood Transplantation In Patients With Hematologic Malignancies Using A Myeloablative Preparative Regimen
N/A
21 Years
Both
Hematologic Malignancies, Disorder Related to Transplantation, Hematopoietic Malignancy
Trial Information
Umbilical Cord Blood Transplantation In Patients With Hematologic Malignancies Using A Myeloablative Preparative Regimen
The primary objectives is to estimate the event-free survival (EFS) at one-year
post-transplant for research participants with high-risk hematologic malignancies undergoing
hematopoietic cell transplantation (HCT) using single unit umbilical cord blood (UCB).
Secondary objectives are:
- Describe the clinical outcome of patients undergoing a double unit UCBT.
- Estimate the incidence and severity of acute and chronic graft versus host disease
(GVHD) of patients enrolled in the research arm.
- Estimate the incidence and time to neutrophil and platelet engraftment among patients
enrolled in the research arm.
- Estimate the incidence of transplant related mortality (TRM) and transplant related
morbidity in the first 100 days after transplantation among patients enrolled in the
research
Exploratory Objectives are:
- Assess the relationship between pre-transplant minimal residual disease (MRD) with
transplant outcomes.
- Record immune reconstitution parameters, including chimerism analysis, quantitative
lymphocyte subsets, T cell receptor excision circle (TREC) and spectratyping.
Immunophenotyping and functional assays of T, B and NK cells and lymphocytes will also
be evaluated.
- Evaluate the determinants of engraftment.
- Characterize the pharmacokinetics of mycophenolate mofetil (MMF).
Inclusion Criteria:
- Age less than or equal to 21 years old.
- Has a partially HLA-matched single or double UCB product
- High-risk hematologic malignancy.
- High risk ALL in CR1, ALL in High risk CR2, ALL in CR3 or subsequent.
- AML in high risk CR1, AML in CR2 or subsequent
- AML in first relapse with < 25% blasts in BM
- Therapy related AML, with prior malignancy in CR > 12mo
- MDS, primary or secondary
- NK cell, biphenotypic, or undifferentiated leukemia in CR1 or subsequent.
- CML in accelerated phase, or in chronic phase with persistent molecular positivity or
intolerance to tyrosine kinase inhibitor.
- Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous HCT, or
unable to mobilize stem cells for autologous HCT.
- Non-Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous HCT, or
unable to mobilize stem cells for autologous HCT.
- JMML
- All patients with evidence of CNS leukemia must be treated and be in CNS CR to be
eligible for study.
Patient must fulfill pre-transplant evaluation:
- Cardiac shortening fraction ≥ 26%.
- Creatinine clearance ≥ 70 ml/min/1.73m2.
- Forced vital capacity (FVC) ≥ 50% of predicted value or pulse oximetry ≥ 92% on room
air.
- Karnofsky (≥ 16 years) or Lansky (<16 years) performance score ≥ 70
- Bilirubin ≤ 2.5 mg/dL.
- Alanine aminotransferase (ALT) ≤ 5 times the upper limit of normal for age.
- Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age.
Exclusion Criteria:
- Patient has a suitable MSD, volunteer MURD, or KIR mismatched haploidentical donor
available in the necessary time for stem cell donation.
- Patient has any other active malignancy other than the one for which HCT is
indicated.
- Patient had a prior allogeneic HCT
- Patient had an autologous HCT within the previous 12 months.
- Patient is pregnant as confirmed by positive serum or urine pregnancy test within 14
days prior to enrollment.
- Patient is lactating
- Patient has Down Syndrome
- Patient has a current uncontrolled bacterial, fungal, or viral infection per the
judgment of the PI.
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Event Free survival at one- year post transplant will be estimated for research participants by using single unit umbilical cord blood
Outcome Description:
Specifically, event free survival is calculated as the difference between date of HCT and min (last follow-up date, date of relapse, date of graft failure, date of death due to any cause, 1 year post-transplant).
Outcome Time Frame:
1 year
Safety Issue:
Yes
Principal Investigator
Mari H Dallas, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
St. Jude Children's Research Hospital
Authority:
United States: Institutional Review Board
Study ID:
UCBT01
NCT ID:
NCT01328496
Start Date:
June 2011
Completion Date:
June 2020
Related Keywords:
- Hematologic Malignancies
- Disorder Related to Transplantation
- Hematopoietic Malignancy
- Hematologic Malignancies
- Umbilical Cord Blood transplantation
- Hematopoietic Cell Transplantation
- Neoplasms
- Hematologic Neoplasms
Name | Location |
|---|---|
| St. Jude Children's Research Hospital | Memphis, Tennessee 38105-2794 |
