Trial Outcomes & Findings for GM-CSF Mouthwash for Preventing and Treating Mucositis in Patients Undergoing Radiation Therapy for Head and Neck Cancer (NCT NCT00293462)

Last Updated: 2021-07-30

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

91 participants

Primary outcome timeframe

From baseline to onset of mucositis, approximately 16 days

Results posted on

2021-07-30

Participant Flow

Adult (\> 18 years of age) with confirmed histopathological diagnosis of head and neck carcinoma were recruited at the University of California, San Francisco Radiation Oncology Clinic, the Helen Diller Comprehensive Cancer Center, Saint Francis Medical Center in San Francisco, and Temple University Radiation Oncology Clinic in Philadelphia from May 2005 to Dec 2009.

Of 532 potential subjects that were screened, 374 were excluded as they did not meet the inclusion criteria, 45 refused to participate and 22 were no-shows (declined radiation therapy), thereby reducing our original target sample population to 91.

Participant milestones

Participant milestones
Measure	Arm I: GM-CSF Group (GG) 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.	Arm II: Salt & Soda Group (SS) 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.	Arm III: Salt & Soda Switched to GM-CSF (SG) 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.
Overall Study STARTED	31	29	31
Overall Study COMPLETED	24	21	19
Overall Study NOT COMPLETED	7	8	12

Reasons for withdrawal

Reasons for withdrawal
Measure	Arm I: GM-CSF Group (GG) 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.	Arm II: Salt & Soda Group (SS) 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.	Arm III: Salt & Soda Switched to GM-CSF (SG) 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.
Overall Study Withdrawal by Subject	4	3	8
Overall Study Death	1	0	0
Overall Study Lost to Follow-up	0	1	0
Overall Study Change Treatment Regimen (ineligible)	1	0	0
Overall Study Hypersensitive Reaction (to Salt & Soda)	0	1	0
Overall Study Refuse Radiation Treatment	1	0	0
Overall Study No Show	0	0	1
Overall Study Went back to his home country	0	0	1
Overall Study Blood clot after chemotherapy	0	0	1
Overall Study Ischemic stroke after chemotherapy	0	0	1
Overall Study Possible Tracheostomy leakage	0	1	0
Overall Study Low radiation dose in oral cavity	0	1	0
Overall Study Physician Decision	0	1	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm I: GM-CSF Group (GG) n=31 Participants Arm I: Patients receive oral sargramostim (GM-CSF) mouthwash, holding it in their mouths and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm II: Salt & Soda Group (SS) n=29 Participants Arm II: Patients receive oral salt and soda mouthwash, holding it and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm III: Salt & Soda Switched to GM-CSF (SG) n=31 Participants Arm II patients are split, with Arm III being switched to GM-CSF when they develop mucositis.	Total n=91 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	25 Participants n=5 Participants	26 Participants n=7 Participants	26 Participants n=5 Participants	77 Participants n=4 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants	14 Participants n=4 Participants
Age, Continuous	53.55 years STANDARD_DEVIATION 13.73 • n=5 Participants	54.69 years STANDARD_DEVIATION 12.22 • n=7 Participants	56.26 years STANDARD_DEVIATION 11.29 • n=5 Participants	54.48 years STANDARD_DEVIATION 12.43 • n=4 Participants
Sex: Female, Male Female	11 Participants n=5 Participants	2 Participants n=7 Participants	11 Participants n=5 Participants	24 Participants n=4 Participants
Sex: Female, Male Male	20 Participants n=5 Participants	27 Participants n=7 Participants	20 Participants n=5 Participants	67 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	4 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	30 Participants n=5 Participants	28 Participants n=7 Participants	27 Participants n=5 Participants	85 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	10 Participants n=5 Participants	6 Participants n=7 Participants	5 Participants n=5 Participants	21 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	4 Participants n=7 Participants	0 Participants n=5 Participants	6 Participants n=4 Participants
Race (NIH/OMB) White	18 Participants n=5 Participants	16 Participants n=7 Participants	22 Participants n=5 Participants	56 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	3 Participants n=7 Participants	0 Participants n=5 Participants	3 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants	3 Participants n=4 Participants

PRIMARY outcome

Timeframe: From baseline to onset of mucositis, approximately 16 days

Population: Intent to treat population consisted of randomized subjects who completed baseline questionnaire and had at least one dose of mouthwash

Outcome measures

Outcome measures
Measure	Arm I: GM-CSF Group (GG) n=28 Participants Arm I: Patients receive oral sargramostim (GM-CSF) mouthwash, holding it in their mouths and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm II: Salt & Soda Group (SS) n=53 Participants Arm II: Patients receive oral salt and soda mouthwash, holding it and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm III: Salt & Soda Switched to GM-CSF (SG) Arm II patients are split, with Arm III being switched to GM-CSF when they develop mucositis.
Comparison of Number of Participants With Grade 1 or 2 Oral Mucositis (GM-CSF and SS Groups Only)	28 participants	46 participants	—

PRIMARY outcome

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

Population: Intent to treat population consisted of randomized subjects who completed baseline questionnaire and had at least one dose of mouthwash

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.

Outcome measures

Outcome measures
Measure	Arm I: GM-CSF Group (GG) n=28 Participants Arm I: Patients receive oral sargramostim (GM-CSF) mouthwash, holding it in their mouths and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm II: Salt & Soda Group (SS) n=21 Participants Arm II: Patients receive oral salt and soda mouthwash, holding it and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm III: Salt & Soda Switched to GM-CSF (SG) n=19 Participants Arm II patients are split, with Arm III being switched to GM-CSF when they develop mucositis.
Comparison of the Mean Number of Days for Mucositis to Heal Across by Group	81.5 days Standard Deviation 63.43	79.4 days Standard Deviation 31.24	77.4 days Standard Deviation 37.40

SECONDARY outcome

Timeframe: Up to 3 months

Population: Of the intent to treat population, subjects who did not fill out the baseline quality of life questionnaires were not included in our analysis.

Quality of life at baseline, during radiotherapy, and once a month for 3 months after radiotherapy. The items on the quality of life questionnaire developed for the study range in scores from 0-10 with higher scores indicating a better quality of life. Scores at all time points were combined to compute one mean score for overall quality of life during the study.

Outcome measures

Outcome measures
Measure	Arm I: GM-CSF Group (GG) n=28 Participants Arm I: Patients receive oral sargramostim (GM-CSF) mouthwash, holding it in their mouths and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm II: Salt & Soda Group (SS) n=25 Participants Arm II: Patients receive oral salt and soda mouthwash, holding it and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm III: Salt & Soda Switched to GM-CSF (SG) n=27 Participants Arm II patients are split, with Arm III being switched to GM-CSF when they develop mucositis.
Comparison of the Combined Mean Score on the Overall Quality of Life Questionnaires by Group	6.91 score on a scale Standard Deviation 1.59	6.80 score on a scale Standard Deviation 1.46	6.77 score on a scale Standard Deviation 1.53

SECONDARY outcome

Timeframe: Up to 3 months

Population: Of the intent to treat population, subjects who did not fill out the baseline Karnofsky functional scales were not included in our analysis. They withdrew from the study after signing the consent forms for personal reasons.

Functional status of participants was measured by administering the KPS which contains items asking about performing daily activities. The scores are grouped in 10s ranging from 100 (Normal, no complaints) to 0 (Dead), with higher scores indicating better functional status. The KPS was administered at baseline, during radiotherapy, and once a month for 3 months after radiation therapy. Scores at all time points were combined to compute one mean to determine overall performance status during the course of the study.

Outcome measures

Outcome measures
Measure	Arm I: GM-CSF Group (GG) n=28 Participants Arm I: Patients receive oral sargramostim (GM-CSF) mouthwash, holding it in their mouths and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm II: Salt & Soda Group (SS) n=25 Participants Arm II: Patients receive oral salt and soda mouthwash, holding it and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm III: Salt & Soda Switched to GM-CSF (SG) n=26 Participants Arm II patients are split, with Arm III being switched to GM-CSF when they develop mucositis.
Comparison of Combined Mean Score on the Karnofsky Performance Status Scale (KPS) by Group	84.52 score on a scale Standard Deviation 12.61	82.93 score on a scale Standard Deviation 10.92	83.49 score on a scale Standard Deviation 12.49

SECONDARY outcome

Timeframe: Up to 3 months

Population: Of the intent to treat population, subjects who did not fill out the baseline pain questionnaires were not included in our analysis. They withdrew from the study after signing the consent forms for personal reasons.

Severity and quality of pain was measured using a pain questionnaire developed for this study with total scores ranging from 0 (no pain) to 10 (most intense pain) with higher scores indicating a greater level of pain. The questionnaire was administered at baseline, during radiotherapy, and once a month for 3 months after radiotherapy. Scores at all time points were combined to compute one mean to determine overall pain intensity score during the course of the study

Outcome measures

Outcome measures
Measure	Arm I: GM-CSF Group (GG) n=27 Participants Arm I: Patients receive oral sargramostim (GM-CSF) mouthwash, holding it in their mouths and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm II: Salt & Soda Group (SS) n=25 Participants Arm II: Patients receive oral salt and soda mouthwash, holding it and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm III: Salt & Soda Switched to GM-CSF (SG) n=27 Participants Arm II patients are split, with Arm III being switched to GM-CSF when they develop mucositis.
Comparison of Combined Mean Score on the Pain Questionnaire by Group	2.15 score on a scale Standard Deviation 2.40	1.72 score on a scale Standard Deviation 2.26	2.15 score on a scale Standard Deviation 2.65

Adverse Events

Arm I: GM-CSF Group (GG)

Serious events: 1 serious events

Other events: 18 other events

Deaths: 1 deaths

Arm II: Salt & Soda Group (SS)

Serious events: 2 serious events

Other events: 26 other events

Deaths: 0 deaths

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

Serious events: 2 serious events

Other events: 27 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Arm I: GM-CSF Group (GG) n=29 participants at risk Arm I: Patients receive oral sargramostim (GM-CSF) mouthwash, holding it in their mouths and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm II: Salt & Soda Group (SS) n=28 participants at risk Arm II: Patients receive oral salt and soda mouthwash, holding it and swallowing it in intervals over 1 hour once daily. Treatment continues during 6-7 weeks of radiotherapy and until the mucositis heals.	Arm III: Salt & Soda Switched to GM-CSF (SG) n=28 participants at risk Arm II patients are split, with Arm III being switched to GM-CSF when they develop mucositis.
General disorders Other	0.00% 0/29 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	0.00% 0/28 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	3.6% 1/28 • Number of events 1 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.
Gastrointestinal disorders Mucositis Oral	3.4% 1/29 • Number of events 1 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	0.00% 0/28 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	0.00% 0/28 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.
Gastrointestinal disorders Lower gastrointestinal hemorrhage	0.00% 0/29 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	3.6% 1/28 • Number of events 1 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	0.00% 0/28 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.
Vascular disorders Thromboembolic event	0.00% 0/29 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	0.00% 0/28 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	3.6% 1/28 • Number of events 1 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.
Immune system disorders Allergic reaction	0.00% 0/29 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	3.6% 1/28 • Number of events 1 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.	0.00% 0/28 • Up to 3 months Serious Adverse Events and adverse events were collected in the safety population which is comprised of all participants who were both randomized and received at least one dose of the study medication. Number of participants at risk represents the number of participants who received study medications.

Other adverse events

Additional Information

Marylin J. Dodd, RN, PhD, FAAN

University of California, San Francisco

Phone: 415-476-2191

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place