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TerminatedNCT03839940MISTUpdated Jan 29, 2025Results posted

Dexamethasone in Reducing Everolimus-Induced Oral Stomatitis in Patients With Cancer

A Phase 3 interventional study of Dexamethasone and Placebo in Malignant Neoplasm, sponsored by Alliance for Clinical Trials in Oncology. Terminated at 319 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-01-29.

Sponsored by Alliance for Clinical Trials in Oncology · Phase 3, Interventional, and Supportive care

Why this study was terminated
Severe lack of accrual
Phase
Phase 3
Study type
Interventional
Enrollment
39
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

This phase III trial studies how well dexamethasone works in reducing everolimus-induced oral stomatitis in patients with cancer. Dexamethasone may help to reduce the everolimus-induced oral stomatitis so as to improve quality of life in cancer patients.

Read the detailed description

PRIMARY OBJECTIVES:

I. To determine if the initiation of dexamethasone at the start of everolimus treatment prevents mTOR inhibitor-associated stomatitis (mIAS)-associated pain, compared to the initiation of placebo.

II. To determine if the initiation of dexamethasone at the start of everolimus treatment will be superior compared to the initiation of placebo in terms of the overall severity of mIAS-associated pain.

SECONDARY OBJECTIVES:

I. To utilize the same measurement method that was reported in the SWISH trial: A combination of a patient reported pain scale, data from a normalcy of diet questionnaire, and clinician grading of stomatitis to determine the incidence of > grade 2 mIAS.

II. To determine if the initiation of dexamethasone at the start of everolimus increases time to development of mouth pain using daily numerical analog scale patient-reported data collection.

III. To assess if quality of life is better when dexamethasone mouth rinse use starts at the same time as everolimus use versus at the time when mouth pain begins.

IV. To investigate if starting dexamethasone mouth rinse concurrent with starting everolimus improves patients' ability to adhere to everolimus therapy.

V. To compare dexamethasone prescription fill rates and timing between patients who received placebo versus study drug at the initiation of everolimus.

Trial Design:

OUTLINE: Patients are randomized to 1 of 2 groups.

GROUP I: Patients receive everolimus orally (PO) once daily (QD) as standard of care and dexamethasone as mouthwash over 2 minutes 4 times per day (QID) for 8 weeks.

GROUP II: Patients receive everolimus PO QD as standard of care and placebo as mouthwash over 2 minutes QID for 8 weeks.

02

Conditions studied

  • Malignant Neoplasm
03

In context

Neoplasms

9,365 studies on the registry are indexed under Neoplasms; 2,489 are open to participants now.

This study's enrollment of 39 is below the median of 50 across 7,253 interventional studies indexed under Neoplasms.

Browse Neoplasms studies →

Lead sponsor

Alliance for Clinical Trials in Oncology is the lead sponsor of 499 studies on the registry; 27 are open to participants now.

Of its 6 completed or terminated interventional studies of FDA-regulated products, 6 (100%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Inclusion Criteria:

  • Current cancer diagnosis, about to receive oral everolimus 10 mg/day with or without an endocrine agent. Patients about to receive everolimus for off label use for any cancer are also eligible.
  • Not currently receiving chemotherapy or any other agent known to cause mucositis or stomatitis. Trastuzumab and ovarian function suppression are allowed.
  • Any prior chemotherapy or other stomatitis/mucositis-causing therapy must be completed at least 2 weeks prior to registration.
  • Not currently suffering from stomatitis/mucositis or mouth ulcers. Patients should not have had any stomatitis or mouth pain for at least 7 days prior to registration.
  • Patients should not receive any other agent which would be considered treatment for stomatitis or impact the primary endpoint.
  • No history of candida infection (thrush) within the last 3 months.
  • Not currently being treated with corticosteroids.
  • No uncontrolled diabetes mellitus, defined by hemoglobin A1C greater than 8%, although A1C is not needed for all patients, hemoglobin (Hgb)A1C \< 8 is required for everyone with diabetes or suspected diabetes.
  • Patients must be able to read and comprehend English. Local translation, including verbal translation of professionals (PROs) is not permitted.
  • Not pregnant and not nursing, because this study involves an investigational agent whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown. Therefore, for women of childbearing potential only, a negative pregnancy test done =\< 7 days prior to registration is required.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2.
05

Study design

Phase
Phase 3
Primary purpose
Supportive care
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
39 participants (actual)

Study arms

  • Experimental
    Group I (dexamethasone)

    Patients receive 10mg oral everolimus daily as standard of care and 10ml dexamethasone oral mouthwash, swished for 2-minutes daily, for 8 weeks.

    Drug: Dexamethasone · Other: Questionnaire · Other: Quality-of-Life Assessment · Drug: Everolimus

  • Placebo comparator
    Group II (placebo)

    Patients receive 10mg oral everolimus daily as standard of care and 10ml placebo oral mouthwash, swished for 2-minutes daily, for 8 weeks.

    Other: Placebo · Other: Questionnaire · Other: Quality-of-Life Assessment · Drug: Everolimus

Interventions

  • DrugDexamethasone

    Given as mouthwash

  • OtherPlacebo

    Given as mouthwash

  • OtherQuestionnaire

    Ancillary studies

  • OtherQuality-of-Life Assessment

    Ancillary studies

  • DrugEverolimus

    Standard of care

06

What researchers measure

Primary outcomes

  1. Incidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale

    The numerical analogue mouth pain score (NAMPS) is a single item measured on a 0 to 10 scale, with 0 corresponding to "No pain" and 10 corresponding to "Pain as bad as it can be." Will compare the incidence rate of any mouth pain (i.e., any score greater than 0) as assessed by the NAMPS between the prevention versus early treatment approaches using a Chi-square test.

    Time frame: Up to 8 weeks

  2. Severity of Mouth Pain Scores

    Average Area Under the Curve per assessment (aAUCpa) of mouth pain scores as measured by NAMPS will be computed for each patient to summarize the sequence of numerical analogue mouth pain scores over the eight week study period. Scores are reported on a 0-100 scale, where 100 = better outcome QOL (i.e. less pain). The aAUCpa is the average of each AUC between each sequential assessment from treatment-initiation to the end of the treatment at 8 weeks post-treatment-initiation and will be compared between the two treatment arms.

    Time frame: Up to 8 weeks

Secondary outcomes

  1. Incidence of Grade > 2 mIAS as Measured by the SWISH Trial

    Original plan for collecting and analyzing this data did not occur due to study termination. Grade 2 mIAS is defined as having at least one of the following criteria:\> 1. Oral intake assessed at 50 on the Normalcy of Diet Scale 2. Patient's reported oral pain (using visual analogue scale 0-10) that meets one of the following: rating of 7 on two consecutive days rating of 8, 9, or 10 on any one day. The incidence of grade 2 mIAS for each treatment arm will be compared by a Chi-square test.

    Time frame: Up to 8 weeks

  2. Incidence of Mouth Pain

    Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with the incidence and of mild (score of 1-3), moderate (score of 4-6) and severe (score of 7-10) mouth pain as measured by the NAMPS. Will be assessed by Chi-square tests.

    Time frame: Up to 8 weeks

  3. Time to Development of Mouth Pain

    Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with the time to development of mild (score of 1-3), moderate (score of 4-6) and severe (score of 7-10) mouth pain as measured by the NAMPS. Will be assessed by Kaplan-Meier and cumulative incidence curves.

    Time frame: Up to 8 weeks

  4. Quality of Life Assessed by Linear Analogue Self-Assessment

    Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with quality of life is by comparing numerical analogue scale scores as measured by the Linear Analogue Self-Assessment. The Linear Analogue Self Assessment for quality of life is measured on a 0 to 10 scale, with 0 corresponding to "Quality of life as bad as it can be" and 10 corresponding to "Quality of life as good as it can be." Will be assessed by independent samples t-tests.

    Time frame: Up to 8 weeks

  5. Mouth/Throat Sore Level of Activity Interference With Daily Activities

    Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with the level of interference with daily activities as measured by the Patient Reported Outcome (PRO) Common Terminology criteria for Adverse Events (CTCAE) mouth/throat item regarding level of interference with daily activities. The PRO-CTCAE mouth/throat item regarding level of interference with daily activities is measured on a Likert 1-5 scale, with 1 corresponding to "Not at all" and 5 corresponding to "Very much." Will be assessed by independent samples t-tests.

    Time frame: Up to 8 weeks

  6. Sleep Level of Activity Interference With Daily Activities

    Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with the level of interference with daily activities as measured by the PRO-CTCAE sleep item regarding level of interference with daily activities. The PRO-CTCAE sleep item regarding level of interference with daily activities is measured on a Likert 1-5 scale, with 1 corresponding to "Not at all" and 5 corresponding to "Very much." Will be assessed by independent samples t-tests.

    Time frame: Up to 8 weeks

  7. Adherence to Everolimus Therapy Dose

    Original plan for collecting and analyzing this data did not occur due to study termination. Will compare mean daily dose of everolimus between the arms. The mean daily dose of everolimus over 8 weeks will be compared between study arms using independent samples t-tests.

    Time frame: Up to 8 weeks

  8. Incidence of Patients Stopping Everolimus Early

    Original plan for collecting and analyzing this data did not occur due to study termination. Will compare the number of patients who stopped everolimus early between the treatment arms. Will be compared using Chi-squared tests.

    Time frame: Up to 8 weeks

  9. Incidence of Patients Reducing Everolimus Dose

    Original plan for collecting and analyzing this data did not occur due to study termination. Will compare the number of patients who reduced their everolimus dose between the treatment arms. Will be compared using Chi-squared tests.

    Time frame: Up to 8 weeks

  10. Dexamethasone Prescription Fill Rates

    Original plan for collecting and analyzing this data did not occur due to study termination. Will compare dexamethasone prescription fill rates between the treatment groups. This will be compared using Chi-squared tests.

    Time frame: Up to 8 weeks

  11. Time to Dexamethasone Prescription Fill

    Original plan for collecting and analyzing this data did not occur due to study termination. Will compare the time to dexamethasone prescription fill between the treatment groups. This will be compared using Kaplan-Meier and cumulative incidence curves.

    Time frame: Up to 8 weeks

  12. Time to Develop Mouth Sores Per Study Arm Assessed by PRO-CTCAE Mouth/Throat Sore Item Regarding Severity

    Original plan for collecting and analyzing this data did not occur due to study termination. The time to develop at least mild, moderate, or severe (including "Very severe") mouth sores per study arm as measured by the PRO-CTCAE mouth-throat sore item regarding severity. The PRO-CTCAE mouth-throat sore item regarding severity is measured on a Likert 1-5 scale, with 1 corresponding to "None" and 5 corresponding to "Very severe". Will be assessed by Kaplan-Meier and cumulative incidence curves.

    Time frame: Up to 8 weeks

  13. Mean Degrees of Patient-reported Mouth Sores Per Study Arm Assessed by NAMPS

    Original plan for collecting and analyzing this data did not occur due to study termination. The mean degrees of patient-reported mouth sores per study arm as measured by the NAMPS will be assessed by independent samples t-tests.

    Time frame: Up to 8 weeks

  14. Median Degree of Patient-reported Mouth Sores Per Study Arm Assessed by NAMPS

    Original plan for collecting and analyzing this data did not occur due to study termination. The median degrees of patient-reported mouth sores per study arm as measured by the NAMPS will be assessed by independent samples t-tests.

    Time frame: Up to 8 weeks

  15. Proportion of Patients Who Experience Grade 2 Stomatitis

    Original plan for collecting and analyzing this data did not occur due to study termination. The proportion of patients who experience clinician-reported grade 2 stomatitis (as measured by the CTCAE and performed at 1 and 2 months post-baseline) per study arm will be assessed by Chi-squared tests.

    Time frame: Up to 2 months

Other outcomes

  1. Incidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale by Sex

    NIH-required analysis. The numerical analogue mouth pain score (NAMPS) is a single item measured on a 0 to 10 scale, with 0 corresponding to "No pain" and 10 corresponding to "Pain as bad as it can be." Will compare the incidence rate of any mouth pain (i.e., any score greater than 0) as assessed by the NAMPS between the prevention versus early treatment approaches using a Chi-square test.

    Time frame: Up to 8 weeks

  2. Incidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale by Race

    NIH-required analysis. The numerical analogue mouth pain score (NAMPS) is a single item measured on a 0 to 10 scale, with 0 corresponding to "No pain" and 10 corresponding to "Pain as bad as it can be." Will compare the incidence rate of any mouth pain (i.e., any score greater than 0) as assessed by the NAMPS between the prevention versus early treatment approaches using a Chi-square test.

    Time frame: Up to 8 weeks

  3. Incidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale by Ethnicity.

    NIH-required analysis. The numerical analogue mouth pain score (NAMPS) is a single item measured on a 0 to 10 scale, with 0 corresponding to "No pain" and 10 corresponding to "Pain as bad as it can be." Will compare the incidence rate of any mouth pain (i.e., any score greater than 0) as assessed by the NAMPS between the prevention versus early treatment approaches using a Chi-square test.

    Time frame: Up to 8 weeks

  4. Severity of Mouth Pain Scores by Sex

    Average Area Under the Curve per assessment (aAUCpa) of mouth pain scores as measured by NAMPS will be computed for each patient to summarize the sequence of numerical analogue mouth pain scores over the eight week study period. Scores are reported on a 0-100 scale, where 100 = better outcome QOL (i.e. less pain). The aAUCpa is the average of each AUC between each sequential assessment from treatment-initiation to the end of the treatment at 8 weeks post-treatment-initiation and will be compared between the two treatment arms.

    Time frame: Up to 8 weeks

  5. Severity of Mouth Pain Scores by Race

    Average Area Under the Curve per assessment (aAUCpa) of mouth pain scores as measured by NAMPS will be computed for each patient to summarize the sequence of numerical analogue mouth pain scores over the eight week study period. Scores are reported on a 0-100 scale, where 100 = better outcome QOL (i.e. less pain). The aAUCpa is the average of each AUC between each sequential assessment from treatment-initiation to the end of the treatment at 8 weeks post-treatment-initiation and will be compared between the two treatment arms.

    Time frame: Up to 8 weeks

  6. Severity of Mouth Pain Scores by Ethnicity

    Average Area Under the Curve per assessment (aAUCpa) of mouth pain scores as measured by NAMPS will be computed for each patient to summarize the sequence of numerical analogue mouth pain scores over the eight week study period. Scores are reported on a 0-100 scale, where 100 = better outcome QOL (i.e. less pain). The aAUCpa is the average of each AUC between each sequential assessment from treatment-initiation to the end of the treatment at 8 weeks post-treatment-initiation and will be compared between the two treatment arms.

    Time frame: Up to 8 weeks

07

Results

Posted Aug 1, 2022

Participant flow

Participant flow — Overall Study
MilestoneGroup I (Dexamethasone)Group II (Placebo)
Started2019
Completed1617
Not completed42
Withdrew: Withdrawal by subject30
Withdrew: Protocol violation01
Withdrew: No post-baseline pain score data11

Outcome measures

PrimaryIncidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale

The numerical analogue mouth pain score (NAMPS) is a single item measured on a 0 to 10 scale, with 0 corresponding to "No pain" and 10 corresponding to "Pain as bad as it can be." Will compare the incidence rate of any mouth pain (i.e., any score greater than 0) as assessed by the NAMPS between the prevention versus early treatment approaches using a Chi-square test.

Time frame:
Up to 8 weeks
Reported as:
Count of participants · Participants
Incidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale
ParticipantsGroup I (Dexamethasone)Group II (Placebo)
No Pain78
Pain99
Statistical analysis
  • Group I (Dexamethasone) vs Group II (Placebo) · Fisher Exact · p = < 0.9999
PrimarySeverity of Mouth Pain Scores

Average Area Under the Curve per assessment (aAUCpa) of mouth pain scores as measured by NAMPS will be computed for each patient to summarize the sequence of numerical analogue mouth pain scores over the eight week study period. Scores are reported on a 0-100 scale, where 100 = better outcome QOL (i.e. less pain). The aAUCpa is the average of each AUC between each sequential assessment from treatment-initiation to the end of the treatment at 8 weeks post-treatment-initiation and will be compared between the two treatment arms.

Time frame:
Up to 8 weeks
Reported as:
Mean · scores on scale * days
Severity of Mouth Pain Scores
scores on scale * daysGroup I (Dexamethasone)Group II (Placebo)
Severity of Mouth Pain Scores11.3 ± 14.834.6 ± 6.86
Statistical analysis
  • Group I (Dexamethasone) vs Group II (Placebo) · Wilcoxon (Mann-Whitney) · p = 0.3346
SecondaryIncidence of Grade > 2 mIAS as Measured by the SWISH Trial

Original plan for collecting and analyzing this data did not occur due to study termination. Grade 2 mIAS is defined as having at least one of the following criteria:\> 1. Oral intake assessed at 50 on the Normalcy of Diet Scale 2. Patient's reported oral pain (using visual analogue scale 0-10) that meets one of the following: rating of 7 on two consecutive days rating of 8, 9, or 10 on any one day. The incidence of grade 2 mIAS for each treatment arm will be compared by a Chi-square test.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryIncidence of Mouth Pain

Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with the incidence and of mild (score of 1-3), moderate (score of 4-6) and severe (score of 7-10) mouth pain as measured by the NAMPS. Will be assessed by Chi-square tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryTime to Development of Mouth Pain

Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with the time to development of mild (score of 1-3), moderate (score of 4-6) and severe (score of 7-10) mouth pain as measured by the NAMPS. Will be assessed by Kaplan-Meier and cumulative incidence curves.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryQuality of Life Assessed by Linear Analogue Self-Assessment

Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with quality of life is by comparing numerical analogue scale scores as measured by the Linear Analogue Self-Assessment. The Linear Analogue Self Assessment for quality of life is measured on a 0 to 10 scale, with 0 corresponding to "Quality of life as bad as it can be" and 10 corresponding to "Quality of life as good as it can be." Will be assessed by independent samples t-tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryMouth/Throat Sore Level of Activity Interference With Daily Activities

Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with the level of interference with daily activities as measured by the Patient Reported Outcome (PRO) Common Terminology criteria for Adverse Events (CTCAE) mouth/throat item regarding level of interference with daily activities. The PRO-CTCAE mouth/throat item regarding level of interference with daily activities is measured on a Likert 1-5 scale, with 1 corresponding to "Not at all" and 5 corresponding to "Very much." Will be assessed by independent samples t-tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondarySleep Level of Activity Interference With Daily Activities

Original plan for collecting and analyzing this data did not occur due to study termination. Will determine if the initiation of dexamethasone at the start of everolimus is associated with the level of interference with daily activities as measured by the PRO-CTCAE sleep item regarding level of interference with daily activities. The PRO-CTCAE sleep item regarding level of interference with daily activities is measured on a Likert 1-5 scale, with 1 corresponding to "Not at all" and 5 corresponding to "Very much." Will be assessed by independent samples t-tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryAdherence to Everolimus Therapy Dose

Original plan for collecting and analyzing this data did not occur due to study termination. Will compare mean daily dose of everolimus between the arms. The mean daily dose of everolimus over 8 weeks will be compared between study arms using independent samples t-tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryIncidence of Patients Stopping Everolimus Early

Original plan for collecting and analyzing this data did not occur due to study termination. Will compare the number of patients who stopped everolimus early between the treatment arms. Will be compared using Chi-squared tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryIncidence of Patients Reducing Everolimus Dose

Original plan for collecting and analyzing this data did not occur due to study termination. Will compare the number of patients who reduced their everolimus dose between the treatment arms. Will be compared using Chi-squared tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryDexamethasone Prescription Fill Rates

Original plan for collecting and analyzing this data did not occur due to study termination. Will compare dexamethasone prescription fill rates between the treatment groups. This will be compared using Chi-squared tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryTime to Dexamethasone Prescription Fill

Original plan for collecting and analyzing this data did not occur due to study termination. Will compare the time to dexamethasone prescription fill between the treatment groups. This will be compared using Kaplan-Meier and cumulative incidence curves.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryTime to Develop Mouth Sores Per Study Arm Assessed by PRO-CTCAE Mouth/Throat Sore Item Regarding Severity

Original plan for collecting and analyzing this data did not occur due to study termination. The time to develop at least mild, moderate, or severe (including "Very severe") mouth sores per study arm as measured by the PRO-CTCAE mouth-throat sore item regarding severity. The PRO-CTCAE mouth-throat sore item regarding severity is measured on a Likert 1-5 scale, with 1 corresponding to "None" and 5 corresponding to "Very severe". Will be assessed by Kaplan-Meier and cumulative incidence curves.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryMean Degrees of Patient-reported Mouth Sores Per Study Arm Assessed by NAMPS

Original plan for collecting and analyzing this data did not occur due to study termination. The mean degrees of patient-reported mouth sores per study arm as measured by the NAMPS will be assessed by independent samples t-tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryMedian Degree of Patient-reported Mouth Sores Per Study Arm Assessed by NAMPS

Original plan for collecting and analyzing this data did not occur due to study termination. The median degrees of patient-reported mouth sores per study arm as measured by the NAMPS will be assessed by independent samples t-tests.

Time frame:
Up to 8 weeks

No measurements were reported for this outcome.

SecondaryProportion of Patients Who Experience Grade 2 Stomatitis

Original plan for collecting and analyzing this data did not occur due to study termination. The proportion of patients who experience clinician-reported grade 2 stomatitis (as measured by the CTCAE and performed at 1 and 2 months post-baseline) per study arm will be assessed by Chi-squared tests.

Time frame:
Up to 2 months

No measurements were reported for this outcome.

Other pre-specifiedIncidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale by Sex

NIH-required analysis. The numerical analogue mouth pain score (NAMPS) is a single item measured on a 0 to 10 scale, with 0 corresponding to "No pain" and 10 corresponding to "Pain as bad as it can be." Will compare the incidence rate of any mouth pain (i.e., any score greater than 0) as assessed by the NAMPS between the prevention versus early treatment approaches using a Chi-square test.

Time frame:
Up to 8 weeks
Reported as:
Count of participants · Participants
Incidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale by Sex
ParticipantsGroup I (Dexamethasone)Group II (Placebo)
Female — No Pain58
Female — Pain77
Male — No Pain20
Male — Pain22
Statistical analysis
  • Group I (Dexamethasone) vs Group II (Placebo) · Fisher Exact · p = < 0.70361
  • Group I (Dexamethasone) vs Group II (Placebo) · Fisher Exact · p = <0.4667
Other pre-specifiedIncidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale by Race

NIH-required analysis. The numerical analogue mouth pain score (NAMPS) is a single item measured on a 0 to 10 scale, with 0 corresponding to "No pain" and 10 corresponding to "Pain as bad as it can be." Will compare the incidence rate of any mouth pain (i.e., any score greater than 0) as assessed by the NAMPS between the prevention versus early treatment approaches using a Chi-square test.

Time frame:
Up to 8 weeks
Reported as:
Count of participants · Participants
Incidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale by Race
ParticipantsGroup I (Dexamethasone)Group II (Placebo)
Black or African American — No Pain10
Black or African American — Pain31
White — No Pain68
White — Pain68
Statistical analysis
  • Group I (Dexamethasone) vs Group II (Placebo) · Fisher Exact · p = <1.0
  • Group I (Dexamethasone) vs Group II (Placebo) · Fisher Exact · p = <1.0
Other pre-specifiedIncidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale by Ethnicity.

NIH-required analysis. The numerical analogue mouth pain score (NAMPS) is a single item measured on a 0 to 10 scale, with 0 corresponding to "No pain" and 10 corresponding to "Pain as bad as it can be." Will compare the incidence rate of any mouth pain (i.e., any score greater than 0) as assessed by the NAMPS between the prevention versus early treatment approaches using a Chi-square test.

Time frame:
Up to 8 weeks
Reported as:
Count of participants · Participants
Incidence Rate of the mTOR Inhibitor-associated Stomatitis (mIAS)-Related Pain Based on Daily Self-reports Via the Numerical Analogue Scale by Ethnicity.
ParticipantsGroup I (Dexamethasone)Group II (Placebo)
Not Hispanic or Latino — No Pain67
Not Hispanic or Latino — Pain99
Other — No Pain11
Other — Pain00
Statistical analysis
  • Group I (Dexamethasone) vs Group II (Placebo) · Fisher Exact · p = <1.0
Other pre-specifiedSeverity of Mouth Pain Scores by Sex

Average Area Under the Curve per assessment (aAUCpa) of mouth pain scores as measured by NAMPS will be computed for each patient to summarize the sequence of numerical analogue mouth pain scores over the eight week study period. Scores are reported on a 0-100 scale, where 100 = better outcome QOL (i.e. less pain). The aAUCpa is the average of each AUC between each sequential assessment from treatment-initiation to the end of the treatment at 8 weeks post-treatment-initiation and will be compared between the two treatment arms.

Time frame:
Up to 8 weeks
Reported as:
Mean · scores on scale * days
Severity of Mouth Pain Scores by Sex
scores on scale * daysGroup I (Dexamethasone)Group II (Placebo)
Female11.4 (0.0 to 47.3)2.7 (0.0 to 15.7)
Male11.0 (0.0 to 33.4)18.8 (17.0 to 20.7)
Statistical analysis
  • Group I (Dexamethasone) vs Group II (Placebo) · Wilcoxon (Mann-Whitney) · p = <0.1701
  • Group I (Dexamethasone) vs Group II (Placebo) · Wilcoxon (Mann-Whitney) · p = <0.4811
Other pre-specifiedSeverity of Mouth Pain Scores by Race

Average Area Under the Curve per assessment (aAUCpa) of mouth pain scores as measured by NAMPS will be computed for each patient to summarize the sequence of numerical analogue mouth pain scores over the eight week study period. Scores are reported on a 0-100 scale, where 100 = better outcome QOL (i.e. less pain). The aAUCpa is the average of each AUC between each sequential assessment from treatment-initiation to the end of the treatment at 8 weeks post-treatment-initiation and will be compared between the two treatment arms.

Time frame:
Up to 8 weeks
Reported as:
Mean · scores on scale * days
Severity of Mouth Pain Scores by Race
scores on scale * daysGroup I (Dexamethasone)Group II (Placebo)
Black or African American9.3 (0.0 to 22.8)7.9 (7.9 to 7.9)
White12.0 (0.0 to 47.3)4.4 (0.0 to 20.7)
Statistical analysis
  • Group I (Dexamethasone) vs Group II (Placebo) · Wilcoxon (Mann-Whitney) · p = <1.0
  • Group I (Dexamethasone) vs Group II (Placebo) · Wilcoxon (Mann-Whitney) · p = <0.5029
Other pre-specifiedSeverity of Mouth Pain Scores by Ethnicity

Average Area Under the Curve per assessment (aAUCpa) of mouth pain scores as measured by NAMPS will be computed for each patient to summarize the sequence of numerical analogue mouth pain scores over the eight week study period. Scores are reported on a 0-100 scale, where 100 = better outcome QOL (i.e. less pain). The aAUCpa is the average of each AUC between each sequential assessment from treatment-initiation to the end of the treatment at 8 weeks post-treatment-initiation and will be compared between the two treatment arms.

Time frame:
Up to 8 weeks
Reported as:
Mean · scores on scale * days
Severity of Mouth Pain Scores by Ethnicity
scores on scale * daysGroup I (Dexamethasone)Group II (Placebo)
Not Hispanic or Latino12.1 (0.0 to 47.3)4.9 (0.0 to 20.7)
Other0.0 (0.0 to 0.0)0.0 (0.0 to 0.0)
Statistical analysis
  • Group I (Dexamethasone) vs Group II (Placebo) · Wilcoxon (Mann-Whitney) · p = <0.2950

Adverse events

Collected over 8 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Group II (Placebo)0/19 (0%)3/18 (16.7%)16/18 (88.9%)
Group I (Dexamethasone)0/20 (0%)2/17 (11.8%)12/17 (70.6%)
Most frequent serious events
Most frequent serious events
EventGroup II (Placebo)Group I (Dexamethasone)
Enterocolitis infectiousInfections and infestations0/181/17
Tumor painNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/181/17
PneumonitisRespiratory, thoracic and mediastinal disorders0/181/17
Sinus tachycardiaCardiac disorders1/180/17
Lower gastrointestinal hemorrhageGastrointestinal disorders1/180/17
Edema limbsGeneral disorders1/180/17
Creatinine increasedInvestigations1/180/17
DehydrationMetabolism and nutrition disorders1/180/17
HyponatremiaMetabolism and nutrition disorders1/180/17
CoughRespiratory, thoracic and mediastinal disorders1/180/17
Most frequent other events
Showing 10 of 34
Most frequent other events
EventGroup II (Placebo)Group I (Dexamethasone)
FatigueGeneral disorders14/189/17
InsomniaPsychiatric disorders9/184/17
Mucositis oralGastrointestinal disorders6/187/17
Oral painGastrointestinal disorders6/187/17
HyperglycemiaMetabolism and nutrition disorders6/186/17
NauseaGastrointestinal disorders3/182/17
AnorexiaMetabolism and nutrition disorders3/180/17
IrritabilityPsychiatric disorders3/182/17
Mucosal infectionInfections and infestations0/182/17
DiarrheaGastrointestinal disorders2/180/17

Baseline characteristics

Age, Continuous
Age, Continuous(years)Group I (Dexamethasone)Group II (Placebo)Total
Mean65.3 ± 10.7165.3 ± 8.4665.3 ± 9.46
Age, Customized
Age, Customized(Participants)Group I (Dexamethasone)Group II (Placebo)Total
Age group — <50 years112
Age group — 50 - 65 years8715
Age group — >65 years7916
Sex: Female, Male
Sex: Female, Male(Participants)Group I (Dexamethasone)Group II (Placebo)Total
Female121527
Male426
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Group I (Dexamethasone)Group II (Placebo)Total
Hispanic or Latino000
Not Hispanic or Latino151631
Unknown or Not Reported112
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Group I (Dexamethasone)Group II (Placebo)Total
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American415
White121628
More than one race000
Unknown or Not Reported000
Cancer Type
Cancer Type(Participants)Group I (Dexamethasone)Group II (Placebo)Total
Breast111223
Other5510
ECOG Performance Status
ECOG Performance Status(Participants)Group I (Dexamethasone)Group II (Placebo)Total
08715
171017
2101
Continuous Patient Reported Mouth-Pain Score
Continuous Patient Reported Mouth-Pain Score(scores on a scale)Group I (Dexamethasone)Group II (Placebo)Total
Mean0 ± 00.1 ± 0.240.03 ± 0.17

1 further baseline measures are reported on the registry.

08

Study locations

319 sites
  • University of Alabama at Birmingham Cancer Center
    Birmingham, Alabama 35233, United States
  • Kingman Regional Medical Center
    Kingman, Arizona 86401, United States
  • Mercy Hospital Fort Smith
    Fort Smith, Arkansas 72903, United States
  • PCR Oncology
    Arroyo Grande, California 93420, United States
  • Epic Care-Dublin
    Dublin, California 94568, United States
  • Bay Area Breast Surgeons Inc
    Emeryville, California 94608, United States
  • Epic Care Partners in Cancer Care
    Emeryville, California 94608, United States
  • Contra Costa Regional Medical Center
    Martinez, California 94553-3156, United States
  • Alta Bates Summit Medical Center - Summit Campus
    Oakland, California 94609, United States
  • Bay Area Tumor Institute
    Oakland, California 94609, United States
  • Epic Care Cyberknife Center
    Walnut Creek, California 94597, United States
  • Holy Cross Hospital
    Fort Lauderdale, Florida 33308, United States
  • Saint Alphonsus Cancer Care Center-Boise
    Boise, Idaho 83706, United States
  • Saint Alphonsus Cancer Care Center-Caldwell
    Caldwell, Idaho 83605, United States
  • Kootenai Medical Center
    Coeur d'Alene, Idaho 83814, United States
  • Walter Knox Memorial Hospital
    Emmett, Idaho 83617, United States
  • Idaho Urologic Institute-Meridian
    Meridian, Idaho 83642, United States
  • Saint Alphonsus Medical Center-Nampa
    Nampa, Idaho 83686, United States
  • Kootenai Cancer Center
    Post Falls, Idaho 83854, United States
  • Kootenai Cancer Clinic
    Sandpoint, Idaho 83864, United States
  • Rush - Copley Medical Center
    Aurora, Illinois 60504, United States
  • Illinois CancerCare-Bloomington
    Bloomington, Illinois 61704, United States
  • Illinois CancerCare-Canton
    Canton, Illinois 61520, United States
  • Memorial Hospital of Carbondale
    Carbondale, Illinois 62902, United States
  • SIH Cancer Institute
    Carterville, Illinois 62918, United States
  • Illinois CancerCare-Carthage
    Carthage, Illinois 62321, United States
  • Centralia Oncology Clinic
    Centralia, Illinois 62801, United States
  • Carle on Vermilion
    Danville, Illinois 61832, United States
  • Cancer Care Specialists of Illinois - Decatur
    Decatur, Illinois 62526, United States
  • Decatur Memorial Hospital
    Decatur, Illinois 62526, United States
  • Illinois CancerCare-Dixon
    Dixon, Illinois 61021, United States
  • Carle Physician Group-Effingham
    Effingham, Illinois 62401, United States
  • Crossroads Cancer Center
    Effingham, Illinois 62401, United States
  • Illinois CancerCare-Eureka
    Eureka, Illinois 61530, United States
  • Illinois CancerCare-Galesburg
    Galesburg, Illinois 61401, United States
  • Western Illinois Cancer Treatment Center
    Galesburg, Illinois 61401, United States
  • Illinois CancerCare-Kewanee Clinic
    Kewanee, Illinois 61443, United States
  • Illinois CancerCare-Macomb
    Macomb, Illinois 61455, United States
  • Carle Physician Group-Mattoon/Charleston
    Mattoon, Illinois 61938, United States
  • Good Samaritan Regional Health Center
    Mount Vernon, Illinois 62864, United States
  • Cancer Care Center of O'Fallon
    O'Fallon, Illinois 62269, United States
  • Illinois CancerCare-Ottawa Clinic
    Ottawa, Illinois 61350, United States
  • Illinois CancerCare-Pekin
    Pekin, Illinois 61554, United States
  • Illinois CancerCare-Peoria
    Peoria, Illinois 61615, United States
  • Methodist Medical Center of Illinois
    Peoria, Illinois 61636, United States
  • Illinois CancerCare-Peru
    Peru, Illinois 61354, United States
  • Valley Radiation Oncology
    Peru, Illinois 61354, United States
  • Illinois CancerCare-Princeton
    Princeton, Illinois 61356, United States
  • Southern Illinois University School of Medicine
    Springfield, Illinois 62702, United States
  • Springfield Clinic
    Springfield, Illinois 62702, United States
  • Memorial Medical Center
    Springfield, Illinois 62781, United States
  • Southwest Illinois Health Services LLP
    Swansea, Illinois 62226, United States
  • Carle Cancer Center
    Urbana, Illinois 61801, United States
  • The Carle Foundation Hospital
    Urbana, Illinois 61801, United States
  • Rush-Copley Healthcare Center
    Yorkville, Illinois 60560, United States
  • Physicians' Clinic of Iowa PC
    Cedar Rapids, Iowa 52402, United States
  • Iowa Methodist Medical Center
    Des Moines, Iowa 50309, United States
  • Medical Oncology and Hematology Associates-Des Moines
    Des Moines, Iowa 50309, United States
  • Broadlawns Medical Center
    Des Moines, Iowa 50314, United States
  • Iowa Lutheran Hospital
    Des Moines, Iowa 50316, United States
  • Trinity Regional Medical Center
    Fort Dodge, Iowa 50501, United States
  • Methodist West Hospital
    West Des Moines, Iowa 50266-7700, United States
  • Ochsner LSU Health Monroe Medical Center
    Monroe, Louisiana 71202, United States
  • LSU Health Sciences Center at Shreveport
    Shreveport, Louisiana 71103, United States
  • Lafayette Family Cancer Center-EMMC
    Brewer, Maine 04412, United States
  • Mercy Medical Center
    Springfield, Massachusetts 01104, United States
  • Saint Joseph Mercy Hospital
    Ann Arbor, Michigan 48106, United States
  • Bronson Battle Creek
    Battle Creek, Michigan 49017, United States
  • IHA Hematology Oncology Consultants-Brighton
    Brighton, Michigan 48114, United States
  • Saint Joseph Mercy Brighton
    Brighton, Michigan 48114, United States
  • IHA Hematology Oncology Consultants-Canton
    Canton, Michigan 48188, United States
  • Saint Joseph Mercy Canton
    Canton, Michigan 48188, United States
  • Caro Cancer Center
    Caro, Michigan 48723, United States
  • IHA Hematology Oncology Consultants-Chelsea
    Chelsea, Michigan 48118, United States
  • Saint Joseph Mercy Chelsea
    Chelsea, Michigan 48118, United States
  • Hematology Oncology Consultants-Clarkston
    Clarkston, Michigan 48346, United States
  • Newland Medical Associates-Clarkston
    Clarkston, Michigan 48346, United States
  • Ascension Saint John Hospital
    Detroit, Michigan 48236, United States
  • Great Lakes Cancer Management Specialists-Doctors Park
    East China Township, Michigan 48054, United States
  • Genesee Cancer and Blood Disease Treatment Center
    Flint, Michigan 48503, United States
  • Genesee Hematology Oncology PC
    Flint, Michigan 48503, United States
  • Genesys Hurley Cancer Institute
    Flint, Michigan 48503, United States
  • Hurley Medical Center
    Flint, Michigan 48503, United States
  • Helen DeVos Children's Hospital at Spectrum Health
    Grand Rapids, Michigan 49503, United States
  • Mercy Health Saint Mary's
    Grand Rapids, Michigan 49503, United States
  • Spectrum Health at Butterworth Campus
    Grand Rapids, Michigan 49503, United States
  • Academic Hematology Oncology Specialists
    Grosse Pointe Woods, Michigan 48236, United States
  • Great Lakes Cancer Management Specialists-Van Elslander Cancer Center
    Grosse Pointe Woods, Michigan 48236, United States
  • Michigan Breast Specialists-Grosse Pointe Woods
    Grosse Pointe Woods, Michigan 48236, United States
  • Bronson Methodist Hospital
    Kalamazoo, Michigan 49007, United States
  • West Michigan Cancer Center
    Kalamazoo, Michigan 49007, United States
  • Borgess Medical Center
    Kalamazoo, Michigan 49048, United States
  • Sparrow Hospital
    Lansing, Michigan 48912, United States
  • Hope Cancer Clinic
    Livonia, Michigan 48154, United States
  • Saint Mary Mercy Hospital
    Livonia, Michigan 48154, United States
  • Great Lakes Cancer Management Specialists-Macomb Medical Campus
    Macomb, Michigan 48044, United States
  • Michigan Breast Specialists-Macomb Township
    Macomb, Michigan 48044, United States
  • Saint Mary's Oncology/Hematology Associates of Marlette
    Marlette, Michigan 48453, United States
  • Mercy Health Mercy Campus
    Muskegon, Michigan 49444, United States
  • Lakeland Hospital Niles
    Niles, Michigan 49120, United States

Showing the first 100 of 319 sites.

09

References and documents

Study documents

  • Protocol and statistical analysis plan · Aug 14, 2020

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Select individual patient-level data from this trial can be requested from the NCTN/NCORP Data Archive

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 29, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03839940
Lead sponsor
Alliance for Clinical Trials in Oncology
Collaborators
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Feb 15, 2019
Start date
Jun 3, 2019
Primary completion
Jan 8, 2021
Completion
Aug 3, 2022
Results posted
Aug 1, 2022
Last update
Jan 29, 2025

Study contacts

Kathryn Ruddy, MD
study chair · Mayo Clinic

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Jan 2025. You cannot join it, but the record below documents what was studied.

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