Study of Epidermal Growth Factor on Oral Mucositis Induced by Intensive Chemotherapy for Hematologic Malignancies

Study Overview
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Brief Summary
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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
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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.

Official Title
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Randomized Phase II Study of Recombinant Human Epidermal Growth Factor (rhEGF) on Oral Mucositis Induced by Intensive Chemotherapy for Hematologic Malignancies

Conditions
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Oral Mucositis

Intervention / Treatment
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* Drug: rhEGF + povidone iodine, chlorhexidine, & nystatin
* Drug: Placebo + povidone iodine, chlorhexidine, & nystatin


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: EGF<br>rhEGF + povidone iodine, chlorhexidine, & nystatin | Drug: rhEGF + povidone iodine, chlorhexidine, & nystatin<br>* Topical application of 50 µg/mL rhEGF solution twice daily + oral gargling with povidone iodine (1%), chlorhexidine (0.5%) & nystatin (5,000IU/mL) three times a day. Application of drugs begins with start of intensive chemotherapy and ends at the time of recovery from neutropenia (ANC>1000/µL for 3 days) or disappearance of oral mucositis. For each application of rhEGF, spray total 6 times at palate, oropharynx, both buccal mucosa, tongue, and gingiva.<br>* Other names: Easyef(R);|
| Placebo Comparator: Placebo<br>Placebo + povidone iodine, chlorhexidine, & nystatin | Drug: Placebo + povidone iodine, chlorhexidine, & nystatin<br>* Topical application of placebo drug twice daily + oral gargling with povidone iodine (1%), chlorhexidine (0.5%), and nystatin (5,000IU/mL) three times a day. Application of drugs begins with start of intensive chemotherapy and ends at the time of recovery from neutropenia (ANC>1000/µL for 3 days) or disappearance of oral mucositis. For each application of placebo drug, spray total 6 times at palate, oropharynx, both buccal mucosa, tongue, and gingiva.<br>* Other names: Placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of oral mucositis of grade 2 or higher (NCI CTCAE 3.0) |  | Assessed daily during application of study drugs | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse events |  | Assessed daily during application of study drugs | 
| Day of onset and duration of oral mucositis of grade 2 or higher (NCI CTCAE 3.0) |  | Assessed daily during application of study drugs | 
| Incidence, day of onset, and duration of oral mucositis of grade 3 or higher (NCI CTCAE 3.0) |  | Assessed daily during application of study drugs | 
| Incidence, day of onset, and duration of oral mucositis of grade 2 or higher (WHO) |  | Assessed daily during application of study drugs | 
| Incidence, day of onset, and duration of oral mucositis of grade 3 or higher (WHO) |  | Assessed daily during application of study drugs | 
| Incidence, day of onset, and duration of oral mucositis of grade 4 or higher (WHO) |  | Assessed daily during application of study drugs | 
| OMDQ (oral mucositis daily questionnaire) score during treatment |  | Assessed daily during application of study drugs | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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oral mucositis, intensive chemotherapy, stem cell transplantation, epidermal growth factor