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Umbilical Cord Blood Transplantation In Patients With Hematologic Malignancies Using A Myeloablative Preparative Regimen


Phase 2
N/A
21 Years
Open (Enrolling)
Both
Hematologic Malignancies, Disorder Related to Transplantation, Hematopoietic Malignancy

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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 HospitalMemphis, Tennessee  38105-2794