Clinical trial NCT00845819: Study of Epidermal Growth Factor on Oral Mucositis Induced by Intensive Chemotherapy for Hematologic Malignancies
Official title: Randomized Phase II Study of Recombinant Human Epidermal Growth Factor (rhEGF) on Oral Mucositis Induced by Intensive Chemotherapy for Hematologic Malignancies
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Oral Mucositis
Keywords: oral mucositis
intensive chemotherapy
stem cell transplantation
epidermal growth factor
Lead sponsor: Seoul National University Hospital
Sponsor class: OTHER
Collaborators: name: Daewoong Pharmaceutical Co. LTD.
First posted: 2009-02-18
Enrollment: 138
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

Brief summary: The purpose of this study is to determine efficacy and toxicity of recombinant human epidermal growth factor (rhEGF) as a preventive drug of oral mucositis during intensive chemotherapy with stem cell transplantation in patients with hematologic malignancies.

Detailed description: Oral mucositis is one of the most common adverse events during chemotherapy and affects quality of life of patients receiving chemotherapy in relation to the dose of drugs. However, there is only one drug (palifermin) approved by the US FDA for the prevention of oral mucositis and the other methods to prevent or treat oral mucositis are just empirical and lack evidences. The results of recent study demonstrated promising efficacy and minimal toxicity of recombinant human epidermal growth factor (rhEGF) as a preventive drug of oral mucositis in head and neck cancer patients undergoing radiotherapy (Wu HG, et al. Cancer 2009;115(16):3699-3708). This clinical trial is a double-blind randomized prospective single-institutional phase II study to evaluate efficacy and toxicity of recombinant human epidermal growth factor (rhEGF) as a preventive drug of oral mucositis during intensive chemotherapy with stem cell transplantation in patients with hematologic malignancies.

Eligibility criteria: Inclusion Criteria:

* Patients with confirmed diagnosis of hematologic malignancies including acute \& chronic leukemia, lymphoma, plasma cell dyscrasia, myelodysplastic syndrome, aplastic anemia, etc.
* Patients who are planned to receive high-dose chemotherapy with SCT
* ECOG performance status 0-2
* Informed consent

Exclusion Criteria:

* Patients having previous history of hypersensitivity to this drug or similar drugs
* Patients having oral ulcer or herpes or severe dental disease at the time of inclusion
* Patients received chemotherapy, radiotherapy, or surgery within 3 weeks
* Patients who had finished clinical trials which could affect the results of this trial within 4 weeks or are attending one at the time of inclusion
* Patients having another diseases which have worse prognosis than patients' hematologic malignancy
* Patients with major psychotic disorder or drug/alcohol abuser
* Women who are pregnant or breastfeeding
* Refusal at patients' will
* Inappropriate patients according to the investigators' opinion

Primary outcomes:
measure: Incidence of oral mucositis of grade 2 or higher (NCI CTCAE 3.0)
timeFrame: Assessed daily during application of study drugs
Secondary outcomes:
measure: Adverse events
timeFrame: Assessed daily during application of study drugs
measure: Day of onset and duration of oral mucositis of grade 2 or higher (NCI CTCAE 3.0)
timeFrame: Assessed daily during application of study drugs
measure: Incidence, day of onset, and duration of oral mucositis of grade 3 or higher (NCI CTCAE 3.0)
timeFrame: Assessed daily during application of study drugs
measure: Incidence, day of onset, and duration of oral mucositis of grade 2 or higher (WHO)
timeFrame: Assessed daily during application of study drugs
measure: Incidence, day of onset, and duration of oral mucositis of grade 3 or higher (WHO)
timeFrame: Assessed daily during application of study drugs
measure: Incidence, day of onset, and duration of oral mucositis of grade 4 or higher (WHO)
timeFrame: Assessed daily during application of study drugs
measure: OMDQ (oral mucositis daily questionnaire) score during treatment
timeFrame: Assessed daily during application of study drugs
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
Study officials:
affiliation: Seoul National University Hospital
name: Sung-Soo Yoon, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Keefe DM, Schubert MM, Elting LS, Sonis ST, Epstein JB, Raber-Durlacher JE, Migliorati CA, McGuire DB, Hutchins RD, Peterson DE; Mucositis Study Section of the Multinational Association of Supportive Care in Cancer and the International Society for Oral Oncology. Updated clinical practice guidelines for the prevention and treatment of mucositis. Cancer. 2007 Mar 1;109(5):820-31. doi: 10.1002/cncr.22484.
citation: Sonis ST, Oster G, Fuchs H, Bellm L, Bradford WZ, Edelsberg J, Hayden V, Eilers J, Epstein JB, LeVeque FG, Miller C, Peterson DE, Schubert MM, Spijkervet FK, Horowitz M. Oral mucositis and the clinical and economic outcomes of hematopoietic stem-cell transplantation. J Clin Oncol. 2001 Apr 15;19(8):2201-5. doi: 10.1200/JCO.2001.19.8.2201.
citation: Brown GL, Curtsinger L 3rd, Brightwell JR, Ackerman DM, Tobin GR, Polk HC Jr, George-Nascimento C, Valenzuela P, Schultz GS. Enhancement of epidermal regeneration by biosynthetic epidermal growth factor. J Exp Med. 1986 May 1;163(5):1319-24. doi: 10.1084/jem.163.5.1319.
citation: Sonis ST, Costa JW Jr, Evitts SM, Lindquist LE, Nicolson M. Effect of epidermal growth factor on ulcerative mucositis in hamsters that receive cancer chemotherapy. Oral Surg Oral Med Oral Pathol. 1992 Dec;74(6):749-55. doi: 10.1016/0030-4220(92)90402-c.
citation: Epstein JB, Gorsky M, Guglietta A, Le N, Sonis ST. The correlation between epidermal growth factor levels in saliva and the severity of oral mucositis during oropharyngeal radiation therapy. Cancer. 2000 Dec 1;89(11):2258-65. doi: 10.1002/1097-0142(20001201)89:113.0.co;2-z.
citation: Hong JP, Lee SW, Song SY, Ahn SD, Shin SS, Choi EK, Kim JH. Recombinant human epidermal growth factor treatment of radiation-induced severe oral mucositis in patients with head and neck malignancies. Eur J Cancer Care (Engl). 2009 Nov;18(6):636-41. doi: 10.1111/j.1365-2354.2008.00971.x. Epub 2009 Apr 23.
citation: Wu HG, Song SY, Kim YS, Oh YT, Lee CG, Keum KC, Ahn YC, Lee SW. Therapeutic effect of recombinant human epidermal growth factor (RhEGF) on mucositis in patients undergoing radiotherapy, with or without chemotherapy, for head and neck cancer: a double-blind placebo-controlled prospective phase 2 multi-institutional clinical trial. Cancer. 2009 Aug 15;115(16):3699-708. doi: 10.1002/cncr.24414.