GM-CSF Mouthwash for Preventing and Treating Mucositis in Patients Undergoing Radiation Therapy for Head and Neck Cancer

NCT00293462 | Completed Phase 3 Results FDA Drug

• 4 other identifiers: CDR0000459510UCSF-H452-26184-03028025R01CA107080
• Study Type: interventional
• Target: 91
• Locations: 1 country
• Sites: 1

Brief Summary

GM-CSF may protect normal cells from the side effects, such as mucositis, of radiation therapy and may help damaged tissue heal faster after radiation therapy. This randomized clinical trial is studying how well GM-CSF works in preventing and treating mucositis in patients who are undergoing radiation therapy for head and neck cancer.

Conditions

Head and Neck Cancer; Mucositis Oral; Radiation Toxicity; Tongue Cancer

Keywords

Recurrent squamous, basal cell carcinoma of the pharynx,larynx,lip; Oropharynx, paranasal sinus and nasal cavity; Stage I-IV lymphoepithelioma of the nasopharynx; Stage I-IV lymphoepithelioma of the oropharynx; Recurrent esthesioneuroblastoma of paranasal sinus, nasal cavity; Recurrent inverted papilloma of the paranasal sinus, nasal cavity; Recurrent midline granuloma of the paranasal sinus, nasal cavity; Untreated, current, metastatic squamous neck cancer; Recurrent verrucous carcinoma of the larynx; Recurrent lymphoepithelioma of the oropharynx; Stage I verrucous carcinoma of the larynx; Recurrent verrucous carcinoma of the oral cavity; Stage I verrucous carcinoma of the oral cavity; Stage II verrucous carcinoma of the larynx; Stage II verrucous carcinoma of the oral cavity; Stage III verrucous carcinoma of the larynx; Stage III verrucous carcinoma of the oral cavity; Stage IV verrucous carcinoma of the larynx; Stage IV verrucous carcinoma of the oral cavity; Recurrent adenoid cystic carcinoma of the oral cavity; Stage I adenoid cystic carcinoma of the oral cavity; Stage II adenoid cystic carcinoma of the oral cavity; Stage III adenoid cystic carcinoma of the oral cavity; Stage IV adenoid cystic carcinoma of the oral cavity; Recurrent mucoepidermoid carcinoma of the oral cavity; Stage I mucoepidermoid carcinoma of the oral cavity; Stage II mucoepidermoid carcinoma of the oral cavity; Stage III mucoepidermoid carcinoma of the oral cavity; Stage IV mucoepidermoid carcinoma of the oral cavity; Recurrent squamous cell carcinoma of the lip and oral cavity; Stage I squamous cell carcinoma of the lip and oral cavity; Stage II squamous cell carcinoma of the lip and oral cavity; Stage III squamous cell carcinoma of the lip and oral cavity; Stage IV squamous cell carcinoma of the lip and oral cavity

Outcome Measures

Primary Outcomes (2)

• Comparison of Number of Participants With Grade 1 or 2 Oral Mucositis (GM-CSF and SS Groups Only)

  The number of participants with recorded grade 1 (mild; Irritation, may experience slight pain, not requiring analgesic) or grade 2 (moderate; Patchy mucositis that may produce inflammatory serosanguinitis discharge; may experience moderate pain requiring analgesia) oral mucositis using the Radiation Therapy Oncology Group (RTOG) Acute Radiation Morbidity Scoring Criteria Oral Mucosa Assessment Scale at baseline and during radiotherapy will be recorded. The RTOG grading is reliant on a clinician's ability to judge the anatomical changes associated with oral mucositis (size and characteristics of ulceration), with symptoms ranging from Grade 0 (no symptoms) to Grade 4 (Ulceration, hemorrhage, or necrosis). A comparison of incidence in the GM-CSF Group (GG) and Arm II: Salt \& Soda Group (SS) will be reported.

  From baseline to onset of mucositis, approximately 16 days

• Comparison of the Mean Number of Days for Mucositis to Heal Across by Group

  The mean number of days for mucositis to heal will be used to evaluate the effectiveness of the two mouthwashes in treating oral mucositis as defined by the incidence of Radiation Therapy Oncology Group Acute Radiation Morbidity Scoring.

  From onset of mucositis to healing of mucositis, approximately 80 days

Secondary Outcomes (3)

• Comparison of the Combined Mean Score on the Overall Quality of Life Questionnaires by Group

  Up to 3 months

• Comparison of Combined Mean Score on the Karnofsky Performance Status Scale (KPS) by Group

  Up to 3 months

• Comparison of Combined Mean Score on the Pain Questionnaire by Group

  Up to 3 months

Study Arms (3)

Arm I: GM-CSF Group (GG)

ACTIVE COMPARATOR

Arm I: Patients were randomized to receive oral sargramostim (GM-CSF) mouthwash as a prevention, holding it in their mouths and swallowing it in intervals over 1 hour once daily. If they develop mucositis, they continue receiving GM-CSF treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.

Biological: sargramostim

Arm II: Salt & Soda Group (SS)

ACTIVE COMPARATOR

Arm II: Patients were randomized to receive salt and soda (SS) mouthwash as a prevention, holding it in their mouths and swallowing it in intervals over 1 hour once daily. If they develop mucositis, they continue receiving SS treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.

Other: oral salt and soda mouthwash

Arm III: Salt & Soda Switched to GM-CSF (SG)

ACTIVE COMPARATOR

Arm III: Patients were randomized to receive oral salt and soda (SS) mouthwash as a prevention, holding it in their mouths and swallowing it in intervals over 1 hour once daily. If they develop mucositis, they continue receiving GM-CSF treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.

Biological: sargramostim; Other: oral salt and soda mouthwash

Interventions

sargramostim BIOLOGICAL

Arm I: GM-CSF Group (GG); Arm III: Salt & Soda Switched to GM-CSF (SG)

oral salt and soda mouthwash OTHER

Patients receive oral salt and soda mouthwash, holding it and swallowing it in intervals over 1 hour once daily.

Arm II: Salt & Soda Group (SS); Arm III: Salt & Soda Switched to GM-CSF (SG)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histopathologically confirmed diagnosis of head and neck cancer
• Scheduled to undergo continuous course of conventional or hyperfractionated radiotherapy or intensity-modulated radiotherapy (IMRT) with or without concurrent chemotherapy
• Planning to receive a total radiation dose ≥ 5,500 centigray (cGy), administered in a single daily fraction of 180-220 cGy (5 days a week) or twice daily fractions of 110-150 cGy
• Normal baseline oral examinations (no pre-existing lesion)
• Karnofsky performance status 60-100%
• Mentally capable of participating in research protocol
• Expected survival \> 4.5 months
• Absolute neutrophil count ≥ 1,500/mm\^3
• Platelet count ≥ 100,000/mm\^3
• Creatinine ≤ 2.0 mg/dL
• Bilirubin ≤ 2.0 mg/dL
• Aspartate aminotransferase (AST) \< 5 times upper limit of normal
• HIV negative

You may not qualify if:

• No unresolved adverse event from previous therapy
• No prior radiotherapy to the head and neck
• No prior or concurrent brachytherapy
• No prior participation in this study
• No T1 or T2 glottic tumors
• No other serious concurrent medical illness
• No history of insulin-dependent diabetes mellitus
• No prior hypersensitivity reaction to yeast material
• No recent history of oral ulceration, herpes simplex, oral candidiasis, severe gingivitis, active or chronic mucositis, or xerostomia
• No current New York Heart Association class II-IV congestive heart failure
• Not pregnant or nursing
• No chemotherapy, radiotherapy, or other investigational drugs within the past 4 weeks
• No major surgery within the past 2 weeks
• No systemic sargramostim (GM-CSF) within the past 7 days
• No systemic filgrastim (G-CSF) within the past 24 hours

Sponsors & Collaborators

• Marilyn Dodd: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

MeSH Terms

Conditions

Head and Neck Neoplasms; Stomatitis; Radiation Injuries; Tongue Neoplasms; Esthesioneuroblastoma, Olfactory

Interventions

sargramostim; Salts

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Mouth Diseases; Stomatognathic Diseases; Wounds and Injuries; Mouth Neoplasms; Tongue Diseases; Neuroblastoma; Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Olfactory Nerve Diseases; Cranial Nerve Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Inorganic Chemicals

Results Point of Contact

• Title: Marylin J. Dodd, RN, PhD, FAAN
• Organization: University of California, San Francisco

Marylin.dodd@ucsf.edu

415-476-2191

Study Officials

• Marylin J. Dodd, RN, PhD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: SUPPORTIVE CARE
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 16, 2006
• First Posted: February 17, 2006
• Study Start: June 7, 2005
• Primary Completion: December 31, 2009
• Study Completion: December 31, 2009
• Last Updated: July 30, 2021
• Results First Posted: May 16, 2013

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)