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The PUMA Trial is a Trial of a Single ProHema Modulated-CB Unit as Part of a Double CB Transplant in Patients With Hematologic Malignancies.

This study has been terminated.
(Business decision)
Information provided by (Responsible Party):
Fate Therapeutics Identifier:
First received: June 21, 2012
Last updated: September 9, 2016
Last verified: October 2015
This study is an open-label randomized, prospectively and historically controlled trial of the safety and efficacy of a single ProHema-CB unit used as part of a double CB transplant following myeloablative or reduced intensity conditioning for subjects age 15-65 years with hematologic malignancies. A maximum of 60 eligible subjects will be enrolled and treated in the trial at approximately 10 centers within the U.S.

Condition Intervention Phase
Hematologic Malignancies
Biological: ProHema-CB
Biological: Untreated CB
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2 Controlled Trial of a Single ProHema®-CB Unit (Ex Vivo Modulated Human Cord Blood) As Part of a Double Umbilical Cord Blood Transplant Following Myeloablative or Reduced Intensity Conditioning For Patients Age 15-65 Years With Hematologic Malignancies.

Further study details as provided by Fate Therapeutics:

Primary Outcome Measures:
  • Neutrophil engraftment/chimerism [ Time Frame: before Day 60 ]
    To determine the rate of neutrophil engraftment after a single ProHema-CB unit is used as part of a double CB transplant.

Secondary Outcome Measures:
  • Neutrophil engraftment [ Time Frame: before Day 180 ]
    To define measures of engraftment, including time to neutrophil engraftment, cumulative incidence of neutrophil engraftment by Day 42, time to platelet engraftment (> 20K and > 50K), cumulative incidence of platelet engraftment, and rates of primary and secondary graft failure

Enrollment: 54
Study Start Date: July 2012
Estimated Study Completion Date: February 2017
Primary Completion Date: July 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ProHema-CB Biological: ProHema-CB
Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood
Active Comparator: Control Arm Biological: Untreated CB
Cord Blood

Detailed Description:
All subjects will receive a myeloablative or reduced intensity conditioning regimen, after which they will receive 2 HLA-matched UCB units. A total of 40 subjects will receive one ProHema-CB as part of a double CB transplant and an additional 20 subjects will be enrolled as concurrent controls. The determination of which CB unit will be the ProHema-CB unit will be made based primarily upon the degree of HLA match.

Ages Eligible for Study:   15 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate. Eligible diseases and stages include:

    • Acute lymphoblastic leukemia (including T lymphoblastic leukemia) in complete remission.
    • Remission is defined as < 5% blasts with no morphological characteristics of acute leukemia in a bone marrow with > 20% cellularity.
    • Acute myelogenous leukemia in high risk first CR or second or subsequent CR.
    • High risk first CR is defined by but is not limited to at least one of the following factors: greater than 1 cycle of induction chemotherapy to achieve CR, prior myelodysplastic syndrome (MDS), presence of Flt3 abnormalities, FAB M6 or M7 subtypes of leukemia, or adverse cytogenetics.
    • Remission is defined as < 5% blasts with no morphological characteristics of acute leukemia (e.g. Auer Rods) in a bone marrow with > 20% cellularity.
    • Biphenotypic/Undifferentiated leukemia in first or subsequent CR (same definition of CR as for ALL/AML).
    • Non-Hodgkin's lymphoma (T-cell, large cell or mantle cell) or Hodgkin's lymphoma in second or subsequent complete remission (CR) or in partial remission (PR) with documented chemosensitivity. In addition, marginal zone lymphoma or follicular lymphoma that has progressed after ≥ 2 therapies (excluding single-agent rituximab). No history of prior myeloablative procedure.
  2. Lack of suitable 5-6/6 HLA-matched related or (if institutional guidelines dictate) suitable 8/8 HLA-A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe.
  3. Age 15-65 years.
  4. Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
  5. Signed IRB approved Informed Consent Form (ICF).

Exclusion Criteria:

  1. History of prior allogeneic transplantation
  2. Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular dysfunction (Ejection fraction < 40%) as measured by gated radionuclide ventriculogram or echocardiogram; active angina pectoris, or uncontrolled hypertension; history of myocardial infarction with depressed ejection fraction.
  3. Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of < 50% of predicted, corrected for hemoglobin.
  4. Renal disease: serum creatinine > 2.0 mg/dl and calculated creatinine clearance < 40 mL/min.
  5. Hepatic disease: serum bilirubin > 2.0 mg/dl (except in the case of Gilbert's syndrome or ongoing hemolytic anemia), SGOT or SGPT > 5 x upper limit of normal.
  6. Neurologic disease: symptomatic leukoencephalopathy, active CNS malignancy or other neuropsychiatric abnormalities believed to preclude transplantation.
  7. HIV antibody.
  8. Uncontrolled infection.
  9. Pregnancy or breast feeding mother.
  10. Inability to comply with the requirements for care after allogeneic stem cell transplantation.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01627314

United States, California
City of Hope
Duarte, California, United States, 91010
United States, Georgia
Emory University-Winship Cancer Institute
Atlanta, Georgia, United States, 30322
United States, Illinois
Loyola University Medical Center
Maywood, Illinois, United States, 60153
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Dana-Farber Cancer Institute- Hematopoietic Stem Cell Transplant Program
Boston, Massachusetts, United States, 02215
Boston Children's Hospital
Boston, Massachusetts, United States
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, New York
Mount Sinai Hospital
New York, New York, United States, 10029
United States, Ohio
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, United States, 43210
United States, Oregon
Oregon Health Sciences
Portland, Oregon, United States, 97239
Sponsors and Collaborators
Fate Therapeutics
Study Director: Samuel Dychter, MD Fate Therapeutics
  More Information

Responsible Party: Fate Therapeutics Identifier: NCT01627314     History of Changes
Other Study ID Numbers: FT1050-03
Study First Received: June 21, 2012
Last Updated: September 9, 2016

Additional relevant MeSH terms:
Neoplasms processed this record on April 27, 2017