Trial Outcomes & Findings for Clobetasol for Oral Graft-Versus-Host Disease (NCT NCT01557517)

Last Updated: 2025-06-15

Results Overview

Mucosal changes were assessed by the Oral Mucositis Rating Scale. The primary endpoint was evaluated using the OMRS. Oral tissue changes are rated on a scale of 0-3 compared with normal oral tissue (0-normal/no change, 1-mild change, 2-moderate change, and 3-severe change). Total score is the sum of all OMRS items with a possible range of 0. Total score is the sum of all OMRS items with a possible range of 0-273. Lower score=more normal oral mucosa. There is no standard definition of response in this field. Definitions we used in this pilot study to grade the response to study intervention are: Progress of 25% of initial score (rounded to the closest number) on the OMRS scale. Completion (PD) is defined as an increase of 25% of initial score (rounded to the closest number). Partial Response (PR) is defined as a decrease Response (CR) is defined as a PR plus a score of 0 on the erythema and ulceration components. Stable Disease (SD) does not meet criteria for progression or response.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

40 participants

Primary outcome timeframe

At 4 weeks on active treatment

Results posted on

2025-06-15

Participant Flow

Participant milestones

Participant milestones
Measure	Clobetasol Followed by Assessment + 2 Weeks Clobetasol Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo Followed by Clobetasol Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.
Overall Study STARTED	19	21
Overall Study COMPLETED	17	17
Overall Study NOT COMPLETED	2	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Clobetasol Followed by Assessment + 2 Weeks Clobetasol Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo Followed by Clobetasol Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.
Overall Study Death	1	0
Overall Study Refused further treatment	0	2
Overall Study Adverse Event	0	2
Overall Study Pt declined before treatment started	1	0

Baseline Characteristics

Clobetasol for Oral Graft-Versus-Host Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Clobetasol n=19 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo n=21 Participants Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Total n=40 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
Age, Categorical Between 18 and 65 years	18 Participants n=5 Participants	17 Participants n=7 Participants	35 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	4 Participants n=7 Participants	5 Participants n=5 Participants
Age, Continuous	44.09 years STANDARD_DEVIATION 14.52 • n=5 Participants	41.4 years STANDARD_DEVIATION 16.53 • n=7 Participants	42.75 years STANDARD_DEVIATION 15.53 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	11 Participants n=7 Participants	17 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants	10 Participants n=7 Participants	23 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized White	15 Participants n=5 Participants	13 Participants n=7 Participants	28 Participants n=5 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants
Race/Ethnicity, Customized Unknown	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Multiple Race	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Mexican, Puerto Rican, Cuban, Central or So. Amer.	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized Not meeting definition for Hispanic or Latino	16 Participants n=5 Participants	18 Participants n=7 Participants	34 Participants n=5 Participants
Region of Enrollment United States	19 Participants n=5 Participants	21 Participants n=7 Participants	40 Participants n=5 Participants

PRIMARY outcome

Timeframe: At 4 weeks on active treatment

Population: 18/19 are analyzed in the Clobetasol arm/group because one participant declined participation before treatment started.

Outcome measures

Outcome measures
Measure	Clobetasol (2-week) n=18 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo (2-week) n=21 Participants Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Combined Group 4-week Data n=31 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day. This group includes patients initially randomized to the clobetasol group as well as the patients initially randomized to placebo, who crossed over to the clobetasol 0.05% oral rinse group after 2 weeks of placebo use and the interim assessment and then completed 4 weeks of clobetasol rinse use.
Percentage of Participants With a Response as Assessed by the 273-point Oral Mucositis Rating Scale (OMRS) Who Received Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-versus-host-disease (cGVHD) During a Four-week Treatment Period Progressive Disease	0 percentage of participants	0 percentage of participants	0 percentage of participants
Percentage of Participants With a Response as Assessed by the 273-point Oral Mucositis Rating Scale (OMRS) Who Received Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-versus-host-disease (cGVHD) During a Four-week Treatment Period Partial Response	72 percentage of participants	9 percentage of participants	72 percentage of participants
Percentage of Participants With a Response as Assessed by the 273-point Oral Mucositis Rating Scale (OMRS) Who Received Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-versus-host-disease (cGVHD) During a Four-week Treatment Period Complete Response	0 percentage of participants	0 percentage of participants	19 percentage of participants
Percentage of Participants With a Response as Assessed by the 273-point Oral Mucositis Rating Scale (OMRS) Who Received Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-versus-host-disease (cGVHD) During a Four-week Treatment Period Stable Disease	28 percentage of participants	91 percentage of participants	9 percentage of participants

SECONDARY outcome

Timeframe: Baseline to Day 14 and baseline to Day 28

Population: 18/19 are analyzed in the Clobetasol grp because one participant declined participation before treatment started. Clobetasol and Placebo grps were not assessed separately at Day 28. Only those pts who completed 4 wks of active clobetasol treatment and did not have an active oral infection at the 4 week timepoint were included in the final analysis.

Oral cavity pain was assessed by an oral cavity specific quality of life questionnaire. Pain was rated based on a 0-10 rating scale on which subjects selected a single integer. 0 = no pain and 10 = worst pain. A negative value indicates an increase in oral pain. A positive value indicates an improvement in patient-perceived oral pain.

Outcome measures

Outcome measures
Measure	Clobetasol (2-week) n=18 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo (2-week) n=21 Participants Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Combined Group 4-week Data n=31 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day. This group includes patients initially randomized to the clobetasol group as well as the patients initially randomized to placebo, who crossed over to the clobetasol 0.05% oral rinse group after 2 weeks of placebo use and the interim assessment and then completed 4 weeks of clobetasol rinse use.
Percent Change in Participants' Raw Score of Oral cGVHD Related Pain on a 0-10 Rating Scale Day 28	NA Percent change Standard Deviation NA Day 28 data for this Clobetasol Arm/Group is collapsed within the Combined Group 4-week data Arm/Group.	NA Percent change Standard Deviation NA Day 28 data for this Placebo Arm/Group is collapsed within the Combined Group 4-week data Arm/Group.	45 Percent change Standard Deviation 146
Percent Change in Participants' Raw Score of Oral cGVHD Related Pain on a 0-10 Rating Scale Day 14	13 Percent change Standard Deviation 116	21 Percent change Standard Deviation 67	31 Percent change Standard Deviation 86

SECONDARY outcome

Timeframe: Baseline and 4 weeks on active treatment

Population: 18/19 are analyzed in the Clobetasol grp because one participant declined participation before treatment started.

Oral cavity sensitivity was assessed by an oral cavity specific quality of life questionnaire. Sensitivity was rated based on a 0-10 rating scale on which subjects selected a single integer. 0 = no sensitivity and 10 = worst sensitivity. A negative value indicates an increase in oral sensitivity. A positive value indicates an improvement in patient-perceived oral sensitivity.

Outcome measures

Outcome measures
Measure	Clobetasol (2-week) n=18 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo (2-week) n=21 Participants Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Combined Group 4-week Data n=31 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day. This group includes patients initially randomized to the clobetasol group as well as the patients initially randomized to placebo, who crossed over to the clobetasol 0.05% oral rinse group after 2 weeks of placebo use and the interim assessment and then completed 4 weeks of clobetasol rinse use.
Percent Change in Participants' Raw Score of Oral cGVHD Related Sensitivity on a 0-10 Rating Scale	39 Percent change Standard Deviation 168	19 Percent change Standard Deviation 62	41 Percent change Standard Deviation 106

SECONDARY outcome

Timeframe: Baseline and 4 weeks on active treatment

Population: 18/19 are analyzed in the Clobetasol grp because one participant declined participation before treatment started.

Oral cavity dryness was assessed by an oral cavity specific quality of life questionnaire. Dryness was rated based on a 0-10 rating scale on which subjects selected a single integer. 0 = no dryness and 10 = worst dryness. A negative value indicates an increase in patient-perceived oral dryness. A positive value indicates an improvement in patient-perceived oral dryness.

Outcome measures

Outcome measures
Measure	Clobetasol (2-week) n=18 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo (2-week) n=21 Participants Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Combined Group 4-week Data n=31 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day. This group includes patients initially randomized to the clobetasol group as well as the patients initially randomized to placebo, who crossed over to the clobetasol 0.05% oral rinse group after 2 weeks of placebo use and the interim assessment and then completed 4 weeks of clobetasol rinse use.
Percent Change in Participants' Raw Score of Oral cGVHD Related Dryness on a 0-10 Rating Scale	8 Percent change Standard Deviation 115	15 Percent change Standard Deviation 229	30 Percent change Standard Deviation 111

SECONDARY outcome

Timeframe: pre-rinse, 15-45 min post-rinse, and 90-120 min post-rinse

Population: Per protocol, pharmacokinetic testing was only done on the first 10 patients in the clobetasol group. We are reporting a calculation based on these 3 time points, not a number that can be reported at 3 discrete times.

Steady-state pharmacokinetics of systemic clobetasol were assessed following a single 2 min oral rinse of 0.05% clobetasol on day 14 (1 patient collected on day 12). A noncompartmental pharmacokinetic assessment was performed using WinNonlin v5 (Pharsight Corp, Mountain View, CA) from three sampling times (pre-rinse, 15-45 min post-rinse, and 90-120 min post-rinse). Plasma concentrations of clobetasol were measured using a newly designed and validated LC-MS/MS assay, with a lower limit of quantification of 0.05 ng/mL. The maximum plasma concentrations (CMAX) were recorded as observed values and the area under the plasma-concentration time curve using all three sampling time points (AUCALL) was calculated using the Linear Trapezoidal rule.

Outcome measures

Outcome measures
Measure	Clobetasol (2-week) n=10 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo (2-week) Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Combined Group 4-week Data Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day. This group includes patients initially randomized to the clobetasol group as well as the patients initially randomized to placebo, who crossed over to the clobetasol 0.05% oral rinse group after 2 weeks of placebo use and the interim assessment and then completed 4 weeks of clobetasol rinse use.
Maximum Plasma Concentration (Cmax) of Clobetasol During Pharmacokinetic Testing	0.818 ng/mL Standard Deviation 0.649	—	—

SECONDARY outcome

Timeframe: Baseline Day 0 and Day 28

Population: 37/40 participants are included in this laboratory assessment as interpretable data was not available for 3 patient samples.

Peripheral blood was drawn at baseline and day 28 of clobetasol rinse use, and clobetasol levels were measured in the blood sample. Plasma concentrations of clobetasol were measured using a validated LC-MS/MS assay with a lower limit of quantification of 0.05 ng/mL. Any values below detectable limit or with no peak were adjusted to 0.

Outcome measures

Outcome measures
Measure	Clobetasol (2-week) n=37 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo (2-week) Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Combined Group 4-week Data Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day. This group includes patients initially randomized to the clobetasol group as well as the patients initially randomized to placebo, who crossed over to the clobetasol 0.05% oral rinse group after 2 weeks of placebo use and the interim assessment and then completed 4 weeks of clobetasol rinse use.
Plasma Concentrations of Clobetasol Mouth Rinse in cGVHD Patients at Baseline (Day 0) and Day 28 Baseline (Day 0)	0.0146 ng/mL Standard Deviation 0.0578	—	—
Plasma Concentrations of Clobetasol Mouth Rinse in cGVHD Patients at Baseline (Day 0) and Day 28 Day 28	0.329 ng/mL Standard Deviation 0.343	—	—

SECONDARY outcome

Timeframe: Date treatment consent signed to date off study, approximately 62 months and 12 days.

Population: 18/19 are analyzed in the Clobetasol arm/group because one participant declined participation before treatment started.

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Outcome measures
Measure	Clobetasol (2-week) n=18 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo (2-week) n=21 Participants Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Combined Group 4-week Data n=21 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day. This group includes patients initially randomized to the clobetasol group as well as the patients initially randomized to placebo, who crossed over to the clobetasol 0.05% oral rinse group after 2 weeks of placebo use and the interim assessment and then completed 4 weeks of clobetasol rinse use.
Count of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)	17 Participants	21 Participants	6 Participants

SECONDARY outcome

Timeframe: pre-rinse, 15-45 min post-rinse, and 90-120 min post-rinse

Outcome measures

Outcome measures
Measure	Clobetasol (2-week) n=10 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo (2-week) Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Combined Group 4-week Data Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day. This group includes patients initially randomized to the clobetasol group as well as the patients initially randomized to placebo, who crossed over to the clobetasol 0.05% oral rinse group after 2 weeks of placebo use and the interim assessment and then completed 4 weeks of clobetasol rinse use.
Time to Maximum Plasma Concentration (Cmax) of Clobetasol	1.27 hours Standard Deviation 0.59	—	—

SECONDARY outcome

Timeframe: pre-rinse, 15-45 min post-rinse, and 90-120 min post-rinse

Population: Per protocol, pharmacokinetics testing was only done on the first 10 patients in the clobetasol group.

Outcome measures

Outcome measures
Measure	Clobetasol (2-week) n=10 Participants Patients will rinse oral cavity with 10cc of clobetasol 0.05% for 2 minutes 3 times a day. Clobetasol Oral Rinse: Cycle= up to 4 weeks Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day.	Placebo (2-week) Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day. Placebo oral rinse: Patients will rinse oral cavity with 10cc of placebo oral rinse for 2 minutes 3 times a day.	Combined Group 4-week Data Patients will rinse oral cavity with 10cc of clobetasol 0.05% oral rinse for 2 minutes 3 times a day. This group includes patients initially randomized to the clobetasol group as well as the patients initially randomized to placebo, who crossed over to the clobetasol 0.05% oral rinse group after 2 weeks of placebo use and the interim assessment and then completed 4 weeks of clobetasol rinse use.
Area Under the Plasma Concentration vs Time Curve for All Time Points	0.974 Hr*ng/mL Standard Deviation 0.729	—	—

Adverse Events

Clobetasol

Serious events: 3 serious events

Other events: 16 other events

Deaths: 1 deaths

Placebo First (During Clobetasol Phase Only)

Serious events: 4 serious events

Other events: 21 other events

Deaths: 0 deaths

Placebo (During 2-week Placebo Phase Only)

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Dr. Najla El Jurdi

National Cancer Institute

Phone: 240-992-4033

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place