Trial Outcomes & Findings for Topical Dexamethasone and Tacrolimus for the Treatment of Oral Chronic Graft-Versus-Host Disease (NCT NCT00686855)
NCT ID: NCT00686855
Last Updated: 2014-03-20
Results Overview
Participants were given a survey at the time of screening and 4 weeks after start of therapy. The participants self-reported three symptoms of oral cGVHD: oral sensitivity, mouth pain, and mouth dryness. Each symptom was given a score ranging from 0-10, with 0 as none and 10 as the worst. Improvement in subjective scores was defined as 3 points or further reduction from pre-treatment to post-treatment assessment.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
46 participants
Primary outcome timeframe
Participants were assessed at Baseline and 4 weeks after start of therapy
Results posted on
2014-03-20
Participant Flow
Participant milestones
| Measure |
Dexamethasone
Dexamethasone : Dexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks
|
Tacrolimus
Tacrolimus Arm Closed to Accrual as of January 2012
Tacrolimus : Tacrolimus elixir taken as an oral rinse four times a day for 4 weeks
|
|---|---|---|
|
Overall Study
STARTED
|
28
|
18
|
|
Overall Study
COMPLETED
|
27
|
17
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Dexamethasone
Dexamethasone : Dexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks
|
Tacrolimus
Tacrolimus Arm Closed to Accrual as of January 2012
Tacrolimus : Tacrolimus elixir taken as an oral rinse four times a day for 4 weeks
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
|
Overall Study
Progressive GVHD
|
0
|
1
|
Baseline Characteristics
Topical Dexamethasone and Tacrolimus for the Treatment of Oral Chronic Graft-Versus-Host Disease
Baseline characteristics by cohort
| Measure |
Dexamethasone
n=28 Participants
Dexamethasone : Dexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks
|
Tacrolimus
n=18 Participants
Tacrolimus Arm Closed to Accrual as of January 2012
Tacrolimus : Tacrolimus elixir taken as an oral rinse four times a day for 4 weeks
|
Total
n=46 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
|
22 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
34 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Age, Continuous
|
53.8 years
STANDARD_DEVIATION 11.5 • n=5 Participants
|
54.9 years
STANDARD_DEVIATION 15.3 • n=7 Participants
|
54.2 years
STANDARD_DEVIATION 13 • n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
28 participants
n=5 Participants
|
18 participants
n=7 Participants
|
46 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Participants were assessed at Baseline and 4 weeks after start of therapyPopulation: Of the 46 participants enrolled on the trial, 36 were deemed evaluable for response.
Participants were given a survey at the time of screening and 4 weeks after start of therapy. The participants self-reported three symptoms of oral cGVHD: oral sensitivity, mouth pain, and mouth dryness. Each symptom was given a score ranging from 0-10, with 0 as none and 10 as the worst. Improvement in subjective scores was defined as 3 points or further reduction from pre-treatment to post-treatment assessment.
Outcome measures
| Measure |
Dexamethasone
n=22 Participants
Dexamethasone : Dexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks
|
Tacrolimus
n=14 Participants
Tacrolimus Arm Closed to Accrual as of January 2012
Tacrolimus : Tacrolimus elixir taken as an oral rinse four times a day for 4 weeks
|
|---|---|---|
|
The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.
Improvement in Oral Sensitivity
|
15 participants
|
3 participants
|
|
The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.
Improvement in Mouth Pain
|
11 participants
|
2 participants
|
|
The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.
Improvement in Mouth Dryness
|
11 participants
|
5 participants
|
|
The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.
Improvement in 1 or more areas
|
17 participants
|
7 participants
|
|
The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.
Improvement in 2 or more areas
|
13 participants
|
2 participants
|
|
The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.
Improvement in all 3 areas
|
7 participants
|
1 participants
|
Adverse Events
Dexamethasone
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Tacrolimus
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Dexamethasone
n=28 participants at risk
Dexamethasone : Dexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks
|
Tacrolimus
n=18 participants at risk
Tacrolimus Arm Closed to Accrual as of January 2012
Tacrolimus : Tacrolimus elixir taken as an oral rinse four times a day for 4 weeks
|
|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma
|
0.00%
0/28 • Adverse events were collected during the four week peroid participants took the study treatment.
|
5.6%
1/18 • Number of events 1 • Adverse events were collected during the four week peroid participants took the study treatment.
|
Other adverse events
| Measure |
Dexamethasone
n=28 participants at risk
Dexamethasone : Dexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks
|
Tacrolimus
n=18 participants at risk
Tacrolimus Arm Closed to Accrual as of January 2012
Tacrolimus : Tacrolimus elixir taken as an oral rinse four times a day for 4 weeks
|
|---|---|---|
|
General disorders
Pain - Oral Cavity
|
3.6%
1/28 • Number of events 1 • Adverse events were collected during the four week peroid participants took the study treatment.
|
0.00%
0/18 • Adverse events were collected during the four week peroid participants took the study treatment.
|
|
Infections and infestations
Infection Gr0-2 neutrophils - oral cavity
|
0.00%
0/28 • Adverse events were collected during the four week peroid participants took the study treatment.
|
5.6%
1/18 • Number of events 1 • Adverse events were collected during the four week peroid participants took the study treatment.
|
Additional Information
Nathaniel S. Treister, DMD, DMSc
Dana-Farber Cancer Institute
Phone: 617-632-2806
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place