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
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Factorial Assignment, Masking: Open Label, Primary Purpose: Treatment
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.
Corey Cutler, MD, MPH, FRCP(C)
Dana-Farber Cancer Institute
United States: Food and Drug Administration
|Dana-Farber Cancer Institute||Boston, Massachusetts 02115|
|Massachusetts General Hospital||Boston, Massachusetts 02114-2617|