Clinical trial NCT00041665: Recombinant Human Keratinocyte Growth Factor to Reduce Oral Mucositis in Hematologic Malignancy Patients Undergoing Peripheral Blood Stem Cell Transplantation After Radiation and High-dose Chemotherapy
Official title: A Phase 3, Randomized, Double-blind, Placebo-controlled Trial of Recombinant Human Keratinocyte Growth Factor for Reduction of Mucositis in Patients With Hematologic Malignancies Undergoing Total Body Irradiation and High-dose Chemotherapy With Autologous PBPC Transplantation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Stomatitis
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2002-07-15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

Brief summary: Radiotherapy and high dose chemotherapy with bone marrow transplantation or peripheral blood stem cell transplantation is frequently used as treatment for patients with cancers of the bone marrow or lymph nodes such as Hodgkin's disease, non-Hodgkin's lymphoma, leukemia or multiple myeloma. A common side effect of the radiotherapy and high dose chemotherapy is mucositis (inflammation of the inside of the mouth and throat resulting in pain and difficulty swallowing). Mucositis is often very severe such that patients receive intravenous nutrition and pain medication in the hospital.

In this study, an investigational recombinant human growth factor called Keratinocyte Growth Factor (rHuKGF) is being evaluated to determine its protective effect on the mucosal tissue and its ability to reduce the mouth and throat soreness.


Eligibility criteria: Inclusion Criteria: \* Patients with: non-Hodgkin's lymphoma, Hodgkin's disease, acute myelogenous leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, chronic lymphocytic leukemia, or multiple myeloma \* Eligible for fractionated total body irradiation (fTBI) plus high-dose chemotherapy followed by autologous PBPC support. \* 18 years of age or older \* Karnofsky performance status greater than or equal to 70% \* Minimum of 1.5 Mio CD34+ cells/kg cryopreserved and to be transplanted \* Informed consent for participation in the study Exclusion Criteria: \* History of, or concurrent cancer other than NHL, Hodgkin's disease, AML, ALL, CML, CLL, multiple myeloma \* Prior bone marrow or peripheral blood stem cell transplantation \* Purged stem cell product \* Currently active infection or oral mucositis \* Congestive heart failure \* Serum creatinine \> 1.5x ULN \* Direct bilirubin \> 1.5x ULN \* Transaminases \> 3x ULN \* Corrected DLCO \< 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 is receiving other investigational agent(s). \* Subject is pregnant (eg, positive human chorionic gonadotropin \[HCG\] test) or is breastfeeding. \* Subject refuses to use adequate contraceptive precautions. \* Known hypersensitivity to any of the products to be administered during dosing, including E coli-derived products. \* Inability to give a truly informed consent.

Primary outcomes:
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Locations:
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
citation: Stiff PJ, Emmanouilides C, Bensinger WI, Gentile T, Blazar B, Shea TC, Lu J, Isitt J, Cesano A, Spielberger R. Palifermin reduces patient-reported mouth and throat soreness and improves patient functioning in the hematopoietic stem-cell transplantation setting. J Clin Oncol. 2006 Nov 20;24(33):5186-93. doi: 10.1200/JCO.2005.02.8340. Epub 2006 Jan 3.
citation: Elting LS, Shih YC, Stiff PJ, Bensinger W, Cantor SB, Cooksley C, Spielberger R, Emmanoulides C. Economic impact of palifermin on the costs of hospitalization for autologous hematopoietic stem-cell transplant: analysis of phase 3 trial results. Biol Blood Marrow Transplant. 2007 Jul;13(7):806-13. doi: 10.1016/j.bbmt.2007.03.004. Epub 2007 Apr 23.
citation: Stiff PJ, Erder H, Bensinger WI, Emmanouilides C, Gentile T, Isitt J, Lu ZJ, Spielberger R. Reliability and validity of a patient self-administered daily questionnaire to assess impact of oral mucositis (OM) on pain and daily functioning in patients undergoing autologous hematopoietic stem cell transplantation (HSCT). Bone Marrow Transplant. 2006 Feb;37(4):393-401. doi: 10.1038/sj.bmt.1705250.
citation: Spielberger R, Stiff P, Bensinger W, Gentile T, Weisdorf D, Kewalramani T, Shea T, Yanovich S, Hansen K, Noga S, McCarty J, LeMaistre CF, Sung EC, Blazar BR, Elhardt D, Chen MG, Emmanouilides C. Palifermin for oral mucositis after intensive therapy for hematologic cancers. N Engl J Med. 2004 Dec 16;351(25):2590-8. doi: 10.1056/NEJMoa040125.