Trial Outcomes & Findings for Dexamethasone to Prevent Oral Chronic Graft-versus-Host Disease (NCT NCT00391170)

Last Updated: 2022-02-10

Results Overview

Participants that developed clinically significant (severity score 3 or higher) oral chronic graft versus host disease (GVHD) within 90 days of initiating treatment. Oral chronic GVHD is defined by Lichen Planus (Oral GVHD) Severity Scale (Thongprasom et al., 2003). Score 5: White striae with erosive area \> 1 cm\^2 Score 4: White striae with erosive area \< 1 cm\^2 Score 3: White striae with erythematous area \> 1 cm\^2 Score 2: White striae with erythematous area \< 1 cm\^2 Score 1: Mild white striae only Score 0: No lesions, normal mucosa

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

62 participants

Primary outcome timeframe

Day 90

Results posted on

2022-02-10

Participant Flow

62 participants were consented. 54 participants started the study and 8 participants were screen failure.

Participant milestones

Participant milestones
Measure	Dexamathasone Oral Rinse in Stem Cell Transplant Participants Dexamethasone 0.01% (0.5mg/5 mL) oral rinse solution in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Placebo Oral Rinse in Stem Cell Transplant Participants Placebo oral rinse in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.
Overall Study STARTED	27	27
Overall Study COMPLETED	21	23
Overall Study NOT COMPLETED	6	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Dexamathasone Oral Rinse in Stem Cell Transplant Participants Dexamethasone 0.01% (0.5mg/5 mL) oral rinse solution in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Placebo Oral Rinse in Stem Cell Transplant Participants Placebo oral rinse in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.
Overall Study Withdrawal by Subject	1	0
Overall Study Adverse Event	3	2
Overall Study Physician Decision	2	2

Baseline Characteristics

Dexamethasone to Prevent Oral Chronic Graft-versus-Host Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Dexamathasone Oral Rinse in Stem Cell Transplant Participants n=27 Participants Dexamethasone 0.01% (0.5mg/5 mL) oral rinse solution in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Placebo Oral Rinse in Stem Cell Transplant Participants n=27 Participants Placebo oral rinse in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Total n=54 Participants Total of all reporting groups
Age, Categorical <=18 years	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	26 Participants n=5 Participants	23 Participants n=7 Participants	49 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants	13 Participants n=7 Participants	24 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants	14 Participants n=7 Participants	30 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	12 Participants n=5 Participants	13 Participants n=7 Participants	25 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=5 Participants	14 Participants n=7 Participants	27 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	6 Participants n=5 Participants	3 Participants n=7 Participants	9 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants	9 Participants n=7 Participants	17 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	9 Participants n=5 Participants	10 Participants n=7 Participants	19 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 90

Population: Stem cell transplant participants who started the dexamethasone oral rinse. An intention to treat analysis will be conducted as the primary analysis.

Outcome measures

Outcome measures
Measure	Dexamethasone Oral Rinse in Stem Cell Transplant Participants n=27 Participants Dexamethasone 0.01% (0.5mg/5 mL) oral rinse solution in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Placebo Oral Rinse in Stem Cell Transplant Participants n=27 Participants Placebo oral rinse in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.
Participants That Developed Severity Score 3 or Higher Oral Chronic Graft Versus Host Disease (GVHD).	7 Participants	4 Participants

SECONDARY outcome

Timeframe: Day 90

Population: Stem cell transplant participants who started and completed the study intervention. Three participant were nonevaluable.

Participants that developed oral chronic graft versus host disease (GVHD) within 90 days of initiating treatment. Oral chronic GVHD is defined by Lichen Planus (Oral GVHD) Severity Scale (Thongprasom et al., 2003). Score 5: White striae with erosive area \> 1 cm\^2 Score 4: White striae with erosive area \< 1 cm\^2 Score 3: White striae with erythematous area \> 1 cm\^2 Score 2: White striae with erythematous area \< 1 cm\^2 Score 1: Mild white striae only Score 0: No lesions, normal mucosa

Outcome measures

Outcome measures
Measure	Dexamethasone Oral Rinse in Stem Cell Transplant Participants n=25 Participants Dexamethasone 0.01% (0.5mg/5 mL) oral rinse solution in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Placebo Oral Rinse in Stem Cell Transplant Participants n=26 Participants Placebo oral rinse in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.
Participants With Chronic Oral Graft Versus Host Disease (GVHD) Based on Severity Scores Score of 0	17 Participants	19 Participants
Participants With Chronic Oral Graft Versus Host Disease (GVHD) Based on Severity Scores Score = 1	1 Participants	1 Participants
Participants With Chronic Oral Graft Versus Host Disease (GVHD) Based on Severity Scores Score = 2	0 Participants	2 Participants
Participants With Chronic Oral Graft Versus Host Disease (GVHD) Based on Severity Scores Score = 3 and >	7 Participants	4 Participants

SECONDARY outcome

Timeframe: 3 months

Population: Stem cell transplant participants who started the dexamethasone oral rinse and completed dexamethasone blood draw at 3 month follow up visit.

Participants with systemic absorption of topical dexamethasone based on plasma dexamethasone level \< 30 ng/dL.

Outcome measures

Outcome measures
Measure	Dexamethasone Oral Rinse in Stem Cell Transplant Participants n=22 Participants Dexamethasone 0.01% (0.5mg/5 mL) oral rinse solution in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Placebo Oral Rinse in Stem Cell Transplant Participants n=23 Participants Placebo oral rinse in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.
Participants With Systemic Absorption of Topical Dexamethasone	1 Participants	0 Participants

SECONDARY outcome

Timeframe: 3 months

Population: Stem cell transplant participants who started the dexamethasone oral rinse and completed serum cortisol levels at 3 months follow up visit.

Number of participants who are not on systemic steroids with suppressed adrenal cortical function after completing study intervention phase (3 month) based on serum morning cortisol levels.

Outcome measures

Outcome measures
Measure	Dexamethasone Oral Rinse in Stem Cell Transplant Participants n=20 Participants Dexamethasone 0.01% (0.5mg/5 mL) oral rinse solution in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Placebo Oral Rinse in Stem Cell Transplant Participants n=19 Participants Placebo oral rinse in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.
Participants With Suppressed Adrenal Cortical Function Due to Topical Dexamethasone	1 Participants	1 Participants

Adverse Events

Dexamethasone Oral Rinse in Stem Cell Transplant Participants

Serious events: 1 serious events

Other events: 13 other events

Deaths: 0 deaths

Placebo Oral Rinse in Stem Cell Transplant Participants

Serious events: 0 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Dexamethasone Oral Rinse in Stem Cell Transplant Participants n=27 participants at risk Dexamethasone 0.01% (0.5mg/5 mL) oral rinse solution in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Placebo Oral Rinse in Stem Cell Transplant Participants n=27 participants at risk Placebo oral rinse in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous cell carcinoma of the oral cavity	3.7% 1/27 • 12 months Adverse events were assessed during clinic visits	0.00% 0/27 • 12 months Adverse events were assessed during clinic visits

Other adverse events

Other adverse events
Measure	Dexamethasone Oral Rinse in Stem Cell Transplant Participants n=27 participants at risk Dexamethasone 0.01% (0.5mg/5 mL) oral rinse solution in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.	Placebo Oral Rinse in Stem Cell Transplant Participants n=27 participants at risk Placebo oral rinse in post allogeneic hematopoietic stem cell transplant participants. Rinse oral cavity three times daily for two minutes with ten milliliters then expectorate. Complete for three months duration.
Immune system disorders Graft versus host disease in gastrointestinal tract	18.5% 5/27 • 12 months Adverse events were assessed during clinic visits	3.7% 1/27 • 12 months Adverse events were assessed during clinic visits
Infections and infestations Upper respiratory tract infection	0.00% 0/27 • 12 months Adverse events were assessed during clinic visits	7.4% 2/27 • 12 months Adverse events were assessed during clinic visits
Musculoskeletal and connective tissue disorders Muscular weakness	0.00% 0/27 • 12 months Adverse events were assessed during clinic visits	11.1% 3/27 • 12 months Adverse events were assessed during clinic visits
Vascular disorders Dizziness	0.00% 0/27 • 12 months Adverse events were assessed during clinic visits	11.1% 3/27 • 12 months Adverse events were assessed during clinic visits
Gastrointestinal disorders Abdominal pain	0.00% 0/27 • 12 months Adverse events were assessed during clinic visits	14.8% 4/27 • 12 months Adverse events were assessed during clinic visits
Gastrointestinal disorders Dry mouth	14.8% 4/27 • 12 months Adverse events were assessed during clinic visits	40.7% 11/27 • 12 months Adverse events were assessed during clinic visits
Gastrointestinal disorders Oral pain	11.1% 3/27 • 12 months Adverse events were assessed during clinic visits	29.6% 8/27 • 12 months Adverse events were assessed during clinic visits
General disorders fatigue	3.7% 1/27 • 12 months Adverse events were assessed during clinic visits	18.5% 5/27 • 12 months Adverse events were assessed during clinic visits

Additional Information

Clara, Joseph

National Heart Lung and Blood Institute

Phone: +1 301 402 2399

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place