Phase 2
Completed N=34
Study of Palifermin (Kepivance) in Persons Undergoing Unrelated Donor Allogeneic Hematopoietic Cell Transplantation
Source: ClinicalTrials.gov NCT02356159 ↗Enrolled (actual)
34
Serious AEs
38.2%
Results posted
Jun 2025
Primary outcomePrimary: Phase II: Estimated Percent of Participants Who Experienced Severe Chronic Graft Versus Host Disease (GVHD) — 6.0 Estimated percent of participants
Summary
Background:
- 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 donors' 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.
Objective:
- To see if high doses of palifermin before chemotherapy are safe, prevent chronic GVHD, and improve immune function after transplant.
Eligibility:
- Adults 18 years of age or older with blood or bone marrow cancer with no HLA-matched sibling donor, but with a HLA-matched unrelated donor.
Description of Research Study:
* 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.
* Have more tests to make sure they can have the transplant, including medical history, physical exam, blood tests, disease specific restaging.
* Get palifermin by intravenous (IV) and conditioning chemotherapy to prepare for hematopoietic stem cell transplantation (HSCT). They will get other drugs; some they will take at least 6 months.
* Participants will get the HSCT.
* After transplant, participants will:
* Be hospitalized at least 3-4 weeks.
* Monitored at least weekly for the first 100 days.
* Stay near District of Columbia (D.C). for approximately 100 days post-transplant.
* After 100 days post-transplant - visit National Institutes of Health (NIH) 5 times the first 2 years, then yearly until 5 years post-transplant.
* Additional tests/procedures may be performed to monitor safety, response to transplant, side effects.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Phase II: Estimated Percent of Participants Who Experienced Severe Chronic Graft Versus Host Disease (GVHD) |
6.0 | — |
| PRIMARY Phase I: Maximum Tolerated Dose (MTD) of Palifermin |
720 | — |
| PRIMARY Phase 1: Number of Participants With a Dose-limiting Toxicity (DLT) |
1 | — |
Eligibility Criteria
- INCLUSION CRITERIA:
Patients meeting below eligibility criteria are eligible to receive suitable disease specific therapy for the purposes of disease control while the donor search takes place
- The patient is greater than or equal to 18 years of age.
- Ability of subject to understand and the willingness to sign a written informed consent document.
- Karnofsky performance score >= 60.
- No suitable HLA matched sibling donor is available, and the patient has one or more potentially suitable HLA matched unrelated donor(s) in the National Marrow Donor Registry or other available registry.
- The evaluation of donors shall be in accordance with existing National Marrow Donor Program (NMDP) Standard Policies and Procedures
- HLA-matched donors are defined by allele matching at HLA-A, -B, -C,
- There is a high likelihood that the patient, in the opinion of the principal investigator (PI) or lead associate investigator (LAI), will meet the research phase eligibility criteria and proceed to transplant after induction phase therapy is completed.
- Diagnosis of hematologic malignancy meeting at least one of the disease status criteria outlined in the table below. Diagnoses must be confirmed by the National Cancer Institute (NCI) laboratory of pathology.
- Recipients with acute myeloid leukemia (AML) in first complete remission (CR1) must have one of the following:
- Adverse cytogenetics (as evaluated by history) as defined as complex karyotype (> 3 abnormalities); inv(3) or t(3;3); t(6;9); t(6;11); monosomy 7; trisomy 8, alone or with an abnormality other than t(8;21), t(9;11), inv(16) or t(16;16); or t(11;19)(q23;p13.1) or adverse-risk per European LeukemiaNet (ELN) 2017 criteria.
- Intermediate-risk disease, such as cytogenetically normal AML (CN-AML) with mutations in FMS-like tyrosine kinase 3 (FLT3), DNA methyl transferase 3A (DMNT3A), or additional sex coombs like 1 (ASXL1) or per ELN 2017 criteria.
- Primary induction failure, defined as failure to achieve CR with primary induction chemotherapy.
- Secondary AML, defined as AML related to antecedent myeloid neoplasm or cytotoxic chemotherapy.
- Hyperleukocytosis, white blood cell (WBC) > 100,000, at diagnosis.
- Recipients with ALL in CR1 must have one of the following:
- Adverse cytogenetics defined as translocations involving t(4;11), t(1;19), t(8;14), 11q23, t(9;22) or bcr-abl rearrangement, Philadelphia chromosomelike (Ph-like ALL), or complex cytogenetic abnormalities.
- Presence of minimal residual disease using multicolor flow cytometry or other analytic technique after primary induction chemotherapy.
- Primary induction failure, defined as failure to achieve complete remission (CR) with primary induction chemotherapy.
- Recipients with myelofibrosis must have at least 2 of the following features, or be Dynamic International Prognostic Scoring System (DIPSS) intermediate-2 or high risk:
- Hemoglobin 10 g/dl with transfusion dependence.
- WBC 30, 000/mm^3 or requires cytoreductive therapy to maintain WBC 50% of the expected value (using United States of America (USA)-Information Technology Service (ITS)-National Institutes of Health (NIH) equation) when corrected for Hgb (DLCO Adj.)
- 24hr creatinine clearance or calculated (using the Cockcroft-Gault formula) creatinine clearance > 60 ml/min/1.73 (induction phase only)
- Left ventricular ejection fraction > 45%
- Serum total bilirubin less than 2.5 mg/dl, and serum alanine aminotransferase (ALT) and aspartate transferase (AST) values less than or equal to 2.5 times the upper limit of normal. Patients with elevations of serum total bilirubin up to 10 mg/dl and/or ALT or AST up to 10 times the upper limit of normal may be considered for participation if such elevations are thought to be due to liver involvement by malignancy. However, in these latter patients, if the bilirubin (BR) level does not decrease to less than or equal to 2.5 mg/dl, or AST/ALT do not decrease to less than or equal to 2.5 times the upper limit of nor
Data sourced from ClinicalTrials.gov (NCT02356159). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.