Clinical trial NCT00189488: Trial to Evaluate Palifermin in the Reduction of Acute Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Allogeneic Marrow/Peripheral Blood Progenitor Cell (PBPC) Transplantation
Official title: A Randomized, Double-blind, Placebo-controlled Trial to Evaluate Palifermin (rHuKGF) in the Reduction of Acute Graft Versus Host Disease in Subjects With Hematologic Malignancies Undergoing Allogeneic Marrow/PBPC Transplantation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Graft Versus Host Disease
Hematologic Malignancies
Keywords: Hematological Malignancies
Graft-versus-host-disease
Oral Mucositis
Allogeneic Transplantation
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
Collaborators: name: Amgen
First posted: 2005-09-19
Enrollment: 155
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The main purpose of this study is to evaluate the effect of palifermin versus placebo in the reduction of severe acute graft versus host disease (GVHD) and severe oral mucositis.


Eligibility criteria: Inclusion Criteria:

* Subjects with hematologic malignancies (including myelodysplastic syndromes \[MDS\]) who are considered eligible for Cyclophosphamide (Cy)/Total Body Irradiation(TBI) +/- Etoposide (VP-16); Total Body Irradiation(TBI)/ Etoposide(VP-16); Melphalan(Mel) / Total Body Irradiation(TBI); Busulfan(Bu)/ Cyclophosphamide(Cy); Busulfan(Bu)/ Melphalan (Mel); or Fludarabine(Flu)/ Melphalan(Mel) conditioning therapy with allogeneic stem cell support
* Subjects with a 6/6 Human Leukocyte Antigen (HLA)-matched family member or unrelated donor who would provide donor marrow/ peripheral progenitor stem cells. \[For unrelated matched donors, molecular typing of class I and class II is mandatory\]
* Karnofsky Performance Status \>= 70%
* 18 years of age or older at time of informed consent
* Before any study-specific procedure, the appropriate written informed consent must be obtained

Exclusion Criteria:

* Cancer other than Non-Hodgkin's lymphoma, Hodgkin's disease, acute myelogenous leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, chronic lymphocytic leukemia, myelodysplastic syndrome or multiple myeloma (except: adequately treated basal cell carcinoma of the skin)
* Prior autologous or allogeneic bone marrow or peripheral blood stem cell transplantation
* Previous use of palifermin
* Current active infection (including human immunodeficiency virus (HIV) and hepatitis) or oral mucositis
* Congestive heart failure as defined by New York Heart Association class III or IV
* Graft T-cell depletion for Graft-versus-host disease (GVHD) prophylaxis
* Inadequate renal function (serum creatinine \> 1.5x the upper limit of normal per the institutional guidelines or clearance \< 40 ml/min adjusted for age)
* Inadequate liver function (total bilirubin \> 1.5x the upper limit of normal, aspartate aminotransferase (AST) \> 3x upper limit of normal and/or alanine aminotransferase (ALT) \> 3x upper limit of normal per the institutional guidelines)
* Inadequate pulmonary function as measured by a corrected DLCO (diffusing capacity of the lung for carbon monoxide lung function test) \<50% of predicted
* Subject is currently enrolled in or has not yet completed at least 30 days since ending other investigational device or drug trial(s), or subject is receiving other investigational agent(s)
* Subject of child-bearing potential is evidently pregnant (e.g. positive human chorionic gonadotropin- HCG test) or is breast feeding during Part A of the study
* Subject or partner of subject is not using or refuses to use adequate contraceptive precautions during Part A of the study
* Subject has known sensitivity to any of the products to be administered during dosing including Escherichia coli-derived products
* Subject was previously randomized into this study
* Subject will not be available for follow-up assessments
* Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and/or to comply with study procedures

Primary outcomes:
measure: Number of Participants With Severe (Grade 3 and 4) Acute Graft Versus Host Disease (GVHD)
timeFrame: From transplant (Day 0) until Day 100
Secondary outcomes:
measure: Number of Participants With Grade 2 to 4 Acute Graft Versus Host Disease (GVHD)
timeFrame: From transplant (Day 0) until Day 100
measure: Number of Participants With Day 11 Methotrexate Graft Versus Host Disease Prophylaxis Administration
timeFrame: Day 11
measure: Number of Participants With Severe (Grade 3 or 4) Oral Mucositis
timeFrame: From transplant (Day 0) until Day 100
measure: Duration of Severe Oral Mucositis (WHO Grade 3 and 4)
timeFrame: From transplant (Day 0) until Day 100
measure: Number of Participants With Parenteral or Transdermal Opioid Analgesic Use
timeFrame: From transplant (Day 0) until Day 100
measure: Duration of Hospitalization
timeFrame: From transplant (Day 0) until Day 100
measure: Area Under the Curve (AUC) of Mouth and Throat Soreness Score
timeFrame: The first day of study drug administration through Day 28.
Other outcomes:
Locations:
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References: