A Phase 2, Controlled Trial, of a Single ProHema CB Unit (ex Vivo Modulated CD34+ Hematopoietic Progenitor Cells, Cord Blood) as Part of a Double Umbilical Cord Blod Transplant Following Myeloablative or Reduced Intensity Conditioning in Patients Age 15-65 Years With Hematologic Malignancies.
15 Years
65 Years
Both
Hematologic Malignancies, Allogeneic Stem Cell Transplantation
Trial Information
A Phase 2, Controlled Trial, of a Single ProHema CB Unit (ex Vivo Modulated CD34+ Hematopoietic Progenitor Cells, Cord Blood) as Part of a Double Umbilical Cord Blod Transplant Following Myeloablative or Reduced Intensity Conditioning in Patients Age 15-65 Years With Hematologic Malignancies.
- On the day of admission to the hospital, two intravenous catheters will be placed in
the large veins in the participant's upper chest underneath the collarbone. The
catheters will remain in place throughout the transplant.
- The chemotherapy portion of the treatment is called "Conditioning Therapy". The
chemotherapy is used to prepare the bone marrow space to receive the transplanted
umbilical cord blood units. It consists of three intravenous medications; fludarabine,
melphalan and antithymocyte globulin or thymoglobulin.
- Starting three days before transplant and every day for 6-9 months after the
transplant, participants will receive the immune suppressive drugs tacrolimus and
sirolimus. These drugs are used to prevent Graft-Vs.-Host-Disease (GVHD), which might
develop as the transplant engrafts.
- After the participant completes the "Conditioning Therapy", they will have their
transplant. The day of the transplant is referred to as Day 0. On the day of the
transplant, each cord blood unit will be thawed and washed in the laboratory and be
given through the central venous catheter. All participants receive 2 cord blood units
2-5 hours apart.
- As part of this research study some cord units will be modulated in the laboratory with
ProHema before it is given to the participant. Two different treatment groups will be
tested. Group 1: will have the first cord blood unit modulated with ProHema; Group 2:
will have no modulation with ProHema.
- Participants will also be treated with antibiotics to prevent and treat infection. They
may also receive transfusions of red blood cells and platelets. To help with
engraftment, they will be given the drug G-CSF (Neupogen) starting five days after the
transplant and until their white blood cells recover.
- After participants leave the hospital, they will be required to come back for
monitoring and routine care very frequently. This is standard after umbilical cord
blood transplantation.
Inclusion Criteria:
- Patients with hematologic malignancies for whom allogeneic stem cell transplantation
is deemed clinically appropriate
- Patient must be ineligible for traditional myeloablative transplantation according to
treating physician
- Lack of 6/6 or 5/6 HLA-matched related, 8/8 HLA-matched unrelated donor, or unrelated
donor not available within a time frame necessary to perform a potentially curative
stem cell transplant
- 15-65 years of age
- ECOG Performance Status 0-2
Exclusion Criteria:
- The following hematologic malignancies are excluded:
- Myelofibrosis unless there has been exposure to cytotoxic chemotherapy for the
treatment of progression to acute myeloid leukemia
- Chronic Myelogenous Leukemia, unless there has been exposure to cytotoxic
chemotherapy for the treatment of blast phase, 3) Aplastic anemia, in the
absence of transformation to Myelodysplastic disorder
- Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular
dysfunction as measured by gated radionucleotide ventriculogram or echocardiogram;
active angina pectoris, or uncontrolled hypertension
- Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic
restrictive lung disease, or corrected DLCO of < 50% of predicted, corrected
hemoglobin
- Renal disease: serum creatinine > 2.0mg/dl
- Hepatic disease: serum bilirubin > 2.0mg/dl (expect in the case of Gilbert's syndrome
or ongoing hemolytic anemia), SGOT or SGPT > 3 x upper limit of normal
- Neurologic disease: symptomatic leukoencephalopathy, active CNS malignancy or other
neuropsychiatric abnormalities believed to preclude transplantation
- HIV antibody
- Uncontrolled infection
- Pregnancy or breast feeding mother
- Inability to comply with the requirements for care after allogeneic stem cell
transplantation
Type of Study:
Interventional
Study Design:
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Factorial Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Determine the safety of ex-vivo 16, 16 dimethyl-prostaglandin E2-expanded umbilical cord blood units when used for transplantation in a reduced intensity, sequential umbilical cord blood transplantation model.
Outcome Time Frame:
2 years
Safety Issue:
Yes
Principal Investigator
Corey Cutler, MD, MPH, FRCP(C)
Investigator Role:
Principal Investigator
Investigator Affiliation:
Dana-Farber Cancer Institute
Authority:
United States: Food and Drug Administration
Study ID:
08-274
NCT ID:
NCT00890500
Start Date:
December 2012
Completion Date:
November 2015
Related Keywords:
- Hematologic Malignancies
- Allogeneic Stem Cell Transplantation
- Neoplasms
- Hematologic Neoplasms
Name | Location |
|---|---|
| Dana-Farber Cancer Institute | Boston, Massachusetts 02115 |
| Massachusetts General Hospital | Boston, Massachusetts 02114-2617 |
