A Phase I/II Open Label, Dose Escalation Study of Palifermin (Kepivance) in Persons Undergoing Unrelated Donor Allogeneic Hematopoietic Cell Transplantation

  • End date
    Jan 1, 2024
  • participants needed
  • sponsor
    National Cancer Institute (NCI)
Updated on 16 September 2022
ct scan
graft versus host disease
hematologic malignancy
blast crisis
bone marrow transplant
multiple myeloma
acute leukemia
flow cytometry
ejection fraction
residual disease
cell transplantation
bone marrow procedure
induction chemotherapy
minimal residual disease
autologous transplantation
secondary acute myeloid leukemia
white blood cells
progressive disease
blood transfusion
chemotherapy regimen
bone lesion
therapeutic agents
hepatitis b core antibody
chromosome abnormality
raeb i


  • In allogeneic stem cell transplantation (SCT), stem cells are taken from a donor and given to a recipient. Sometimes the recipient s immune system destroys the donor s cells. Or donor immune cells attack the recipient s tissues, called graft-versus-host disease (GVHD). This is less likely when the recipient and donor have similar human leukocyte antigens (HLA). Researchers want to see if the drug palifermin improves the results of allogeneic SCT from HLA-matched unrelated donors.
  • To see if high doses of palifermin before chemotherapy are safe, prevent chronic GVHD, and improve immune function after transplant.
  • Adults 18 years of age or older with blood or bone marrow cancer with no HLA-matched sibling, but with a possible HLA-matched donor.
  • Participants will be screened with medical history, physical exam, and blood and urine tests. They will have scans and heart and lung exams.
  • Before transplant, participants will:
  • Have many tests and exams. These include blood tests throughout the study and bone marrow biopsy.
  • Get a central line catheter if they do not have one.
  • Have 1-3 rounds of chemotherapy.
  • Take more tests to make sure they can have the transplant, including medical history, physical exam, and CT scan.
  • Get palifermin by IV and more chemotherapy. They will get other drugs, some they will take for 6 months.
  • Participants will get the SCT.
  • After transplant, participants will:
  • Be hospitalized at least 3-4 weeks.
  • Have tests for GVHD at 60 days and 6 months. These include mouth and skin photos and biopsies.
  • Stay near D.C. for 3 months.
  • Visit NIH 5 times the first 2 years, then yearly. They may have scans and biopsies.


  • Graft versus host disease (GVHD) and impaired immune reconstitution are major transplant complications and barriers to improving outcomes after allogeneic hematopoietic stem cell transplantation (alloHSCT) for hematologic malignancies. GVHD is initiated when donor T-cells become alloreactive against recipient major or minor histocompatibility antigens. This process may be exacerbated during the transplantation process by exposure of tissue antigens to donor T-lymphocytes after chemotherapy-induced injury.
  • Palifermin, a recombinant keratinocyte growth factor-1 (KGF-1), imitates the actions of intrinsic KGF and binds to the FGF receptor 2b, which is expressed in the epidermis, oral mucosa, GI mucosa and urothelium, thereby increasing the regenerative capacity of these tissues. Palifermin has been shown to reduce the duration and severity of oral mucositis after intensive chemo-radiotherapy and autologous HSCT for hematologic cancers and is FDA approved. In pre-clinical studies, palifermin has been shown to have an effect on control of acute or chronic GVHD and immune reconstitution after alloHSCT. However, subsequent clinical studies in alloHSCT indicate that the dose and schedule of palifermin as currently

used in humans does not optimize its activity in terms of prevention of GVHD or thymus recovery following alloHSCT.

  • We hypothesize that higher doses of palifermin in the immediate pre alloHSCT conditioning setting will lead to enhanced thymopoiesis, decreased chronic GVHD, and improved immune reconstitution. A dose escalation study is necessary to determine safe dosing levels in persons undergoing alloHSCT.
  • The primary objective of the phase I portion is to assess the safety and tolerability of the administration of the recombinant keratinocyte growth factor (KGF) palifermin in alloHSCT using unrelated donor peripheral blood stem cells.
  • The primary objective of the phase II portion is to determine the incidence of severe chronic GVHD after the addition of palifermin to TMS (tacrolimus, methotrexate and sirolimus) based GVHD prophylaxis delivered in the identical fashion to the NCI 07-C-0195 study.
  • Adults (greater than or equal to 18 years) with advanced or high risk hematologic malignancies (including AML, ALL, MDS, CLL, NHL, HL, CML, multiple myeloma, and MPN)who lack a suitable HLA-matched sibling donor.
  • An unrelated donor matched at a minimum of 8 alleles (HLA-A,-B,-C, and DRB1) by high-resolution typing, identified through the National Marrow Donor Program.
  • Karnofsky greater than or equal to 60 and acceptable organ functions.
  • Patients will receive disease-specific induction chemotherapy (EPOCH-F/R or FLAG) prior to transplant as needed for disease control and immune depletion.
  • All patients will receive an identical conditioning regimen consisting of cyclophosphamide 1200 mg/m^2/day IV for 4 days and fludarabine 30 mg/m^2/day for 4 days (transplant days -6 to -3).
  • All patients will receive a peripheral blood stem cell product from an unrelated donor matched at HLA-A, -B, -C, -DRB1 (8/8) by high-resolution typing.
  • Palifermin will be administered in a phase 1, open label design with the following proposed schedule:
  • Dose level 1: 180 mcg/kg on day -7
  • Dose level 2: 360 mcg/kg on day -7
  • Dose level 3: 540 mcg/kg on day -7
  • Dose level 4: 720 mcg/kg on day -7
  • The phase I portion will be conducted in a standard 3+3 design; the maximum possible number of patients accrued to this portion will be 24.
  • The maximum tolerated dose (MTD) from the phase I portion of the study will be used to conduct a phase II study. Total accrual on the phase II study will be 27 patients, including 3-6 patients treated at the MTD in the phase I portion of the study

Condition Myelodysplastic Syndrome, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Acute Leukemia, Multiple Myeloma
Treatment Rituximab, TMS, Palifermin, Conditioning Chemotherapy, FLAG, EPOCH-F, Hematopoietic Stem Cell Transplant
Clinical Study IdentifierNCT02356159
SponsorNational Cancer Institute (NCI)
Last Modified on16 September 2022


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