Conditions: Lichen Planus
Keywords: Oral lichen planus Dexamethasone solution
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 24
Healthy volunteers: no

Brief summary:
The objective of this single center, 4-week, open label randomized, phase II study is to evaluate and characterize the tolerability and clinical effectiveness of dexamethasone 0.5mg/5ml solution in a mucoadhesive vehicle (Mucolox™) for the treatment of oral lichen planus.

Detailed description:
Potentially eligible subjects will be screened by one of the investigators by asking patients to rate their worst oral pain or pain score (0-10) over the previous week (see Appendix). Those answering with at least a score of "7" (1/10) and that meet all other eligibility requirements (see Section 3) will be eligible for enrollment. All patients will sign informed consent for study participation. Each study visit is anticipated to take approximately 45 minutes.

Treatment will be administered on an outpatient basis. Study medication will be prescribed by authorized study staff physicians at no expense for the patient.

Subjects will be evaluated clinically at baseline before starting treatment and at the end of the four-week period, for a total of two visits. Comprehensive subjective and objective data will be collected and intraoral photographs will be obtained. Oral mucosal disease will be evaluated using both patient reported (questions/visual analog scales) and clinician assessed measures.

Subjects will be prescribed compound dexamethasone 0.5mg/5ml solution in Mucolox™ (ARM A) or dexamethasone 0.5mg/5ml solution only (ARM B). All subjects will also receive a prescription for fluconazole 200 mg tablets once-a-week as prophylactic antifungal therapy. Any subjects that are already taking an antifungal oral medication at the time of the study enrollment will continue their prescribed medication and will not need to take the additional weekly fluconazole dose. Subjects will return for evaluation after four weeks at which time the study end-points will be assessed.

If there is worsening of oral lichen planus that requires initiation of new immunomodulatory medications (systemic or topical), patients will remain on treatment, but will be regarded as unevaluable for the primary endpoint.

Arms:
arm 1: Dexamethasone 0.5 mg/mL, 5ml TID for 4 weeks arm 2: Compounded dexamethasone 0.5 mg/mL in Mucolox™, 5ml TID for 4 weeks

Interventions: intervention 1: Dexamethasone
Intervention descriptions: intervention 1: Dexamethasone solution 0.5 mg/mL, rinse for 5 minutes and expectorate

Protocol document:
Prot_SAP_001.pdf: 
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1 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
NCT02850601 
Dexamethasone Solution for the Treatment of Oral Lichen Planus 
Date : 06/21/2017 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


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Local Protocol #: 
2016P-00164   
 
Version Date:            06/21/17 
 
 
 
TITLE:  
Dexamethasone solution and Dexamethasone in Mucolox™ for the 
Treatment of Oral Lichen Planus 
 
Center: 
 
 
 
 
 
 
Brigham and Women’s Hospital  
 
 
 
 
 
 
 
 
 
75 Francis Street 
 
 
 
 
 
 
 
 
 
Boston, MA 02115 
 
Principal Investigator (PI): 
Alessandro Villa, DDS, PhD, MPH 
 
Brigham and Women’s Hospital  
 
1620 Tremont Street, Suite BC-3-028, Boston, MA 02120 
 
Phone: 617-732-5517 
 
Fax: 617-232-8970 
 
Email: avilla@partners.org 
 
 
 
  
 
 
Other Investigators:  Nathaniel S. Treister, DMD, DMSc; Sook-Bin Woo, DMD, MMSc; Elizabeth 
Waring, DMD; Roxanne Bavarian, DMD; Maryam Jessri, DMD, PhD; Vidya Sankar, DMD, MHS; 
Muhammad Ali Shazib, DMD; Revathi Shekar, DMD; Herve Sroussi, DMD, PhD  
 
Research Assistant: Lisa Bennett Johnson, RDH, MS, MPH 
 
  
Responsible Research Nurse: N/A 
 
Non-study staff: Ms. Casey Argyrou 
 
 
 
Funding: Professional Compounding Centers of America (Contact: Gus Bassani, Pharm.D.) 
 
Agent(s): Mucolox™ (PCCA, Houston, TX 77099); dexamethasone 0.5mg/5mL solution (NDC 00054-
3177-57; West-Ward pharmaceuticals Corp., Eatontown, NJ 07724); dexamethasone sodium phosphate 
USP powder (PCCA, Houston, TX, 77099, part# 55-1430, CAS# 2392-39-4) 
 
 
 
 
 
 
 


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TABLE OF CONTENTS 
1. 
BACKGROUND AND SIGNIFICANCE 
6 
1.1 
Oral lichen planus 
6 
1.2 
Dexamethasone solution 
7 
1.3 
Mucolox™ 
6 
1.4 
Significance 
8 
2. 
SPECIFIC AIMS 
8 
2.1 
Study Design 
8 
2.2 
Primary Objective 
8 
2.3 
Secondary Objective 
8 
2.4 
Hypothesis 
9 
2.5 
Specific Aim 
9 
In order to evaluate the hypothesis, the specific aim for this study is: 
9 
3. 
SUBJECT SELECTION 
9 
3.1 
Eligibility Criteria 
9 
3.2 
Exclusion Criteria 
9 
3.3 
Inclusion of Women and Minorities 
10 
4. 
SUBJECT ENROLLMENT 
10 
     4.1 Procedures for obtaining informed consent 
10 
4.2 
Treatment assignment and randomization 
10 
5. 
STUDY PROCEDURES 
10 
5.2 
Treatment Regimen 
12 
5.3 
Mucolox™ and dexamethasone solution Administration 
13 
5.4 
Dexamethasone solution Administration 
12 
5.5 
Drug Accountability 
13 
5.6 
General Concomitant Medication and Supportive Care Guidelines 
13 
5.7 
Criteria for Taking a Participant Off Protocol Therapy 
14 
5.8 
Duration of Follow Up 
14 
5.9 
Criteria for Taking a Participant Off Study 
15 
6. 
BIOSTATISTICAL ANALYSIS 
15 
6.1 
Study Design/Endpoints 
15 
6.2 
Sample Size, Accrual Rate and Study Duration 
15 
6.3 
Stratification Factors 
14 
6.4 
Interim Monitoring Plan 
15 
6.5 
Analysis of Primary Endpoints 
16 
6.6 
Analysis of Secondary Endpoints 
16 
6.7 
Reporting and Exclusions 
17 
7. 
DOSING DELAYS/DOSE MODIFICATIONS 
17 


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8. 
Risks and discomforts 
17 
8.1 
Drug side effects and toxicities 
17 
9. 
Potential benefits 
18 
9.1 
Potential benefits for participating individuals 
18 
9.2 
Potential benefits to society 
18 
10. 
Monitoring and quality assurance 
18 
10.1 
Safety monitoring 
18 
10.2 
Adverse events 
18 
10.3 
Reported and/or potential AE 
19 
10.4 
Expected Toxicities 
19 
10.5 
Adverse Event Characteristics 
19 
10.6 
Expedited Adverse Event Reporting 
19 
10.7 
Expedited Reporting to the Food and Drug Administration (FDA) 
19 
10.8 
Routine Adverse Event Reporting 
20 
11. 
PHARMACEUTICAL INFORMATION 
20 
11.1 
Mucolox™ 
18 
11.2 
Dexamethasone solution 
22 
12. 
DATA REPORTING / REGULATORY REQUIREMENTS 
23 
12.1 
Monitoring and quality assurance 
23 
13. 
PUBLICATION PLAN 
24 
APPENDICES 
28 
Appendix 1: 
Patient instructions 
28 
Appendix 2: 
Drug diary 
28 
Appendix 3: 
Visit #1 CRF 
28 
Appendix 4: 
Visit #2 CRF 
28 
Appendix 5:  
COMDQLQ 
28 
 
 
   


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SCHEMA 
 
 
 


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1. BACKGROUND AND SIGNIFICANCE 
 
1.1 
Oral lichen planus   
 
Oral lichen planus (OLP) is a common benign, chronic immune-mediated inflammatory condition that 
occurs in 1 to 2% of adults1. Middle-aged women are twice as likely to be affected as men. OLP may 
be associated with a variety of systemic and local conditions and medication use such as thiazide 
diuretics, angiotensin converting enzyme (ACE) inhibitors, beta blockers, gold salts, sulfasalazine, 
sulfonylureas and penicillamine. Biologic agents such as tumor necrosis factor (TNF) alpha inhibitors 
may also cause lichen planus-like eruptions2. Amalgam and composite restorations may cause localized 
contact lichenoid hypersensitivity reactions possibly to mercury and formaldehyde.  
OLP can affect any mucosal surface, and most commonly affects the buccal mucosa and tongue 
bilaterally. OLP can present with three distinct forms: reticular/keratotic, erosive/erythematous, and 
ulcerative forms. The reticular/keratotic form, characterized by Wickham striae, is the most common 
form and is often asymptomatic. Occasionally patients will report discomfort and describe the buccal 
mucosa as “rough,” “thick,” or “tight”. The erosive/erythematous form typically presents as 
desquamative gingivitis. Patients may complain of discomfort while eating acidic, spicy or crunchy 
food. The ulcerative form is the most severe and presents as shallow ulcerations that have a yellow 
fibrin membrane on the surface. Often, there is a combination of the three forms at different sites, or at 
different times. Most patients exhibit a characteristic bilateral and symmetric distribution of lesions, 
typically involving the buccal mucosa, dorsum and ventral surfaces of the tongue and/or gingiva.  
 
The diagnosis of oral lichen planus is based primarily on clinical characteristics, although biopsy may 
be indicated when the presentation is not typical (e.g. unilateral presentation, or lack of reticulation 
features)3,4. Histopathological examination demonstrates a lymphocytic band of variable thickness at 
the interface and degeneration or loss of the basal cell layer.  
 
1.1.1 Management of oral lichen planus 
 
Available therapies for OLP are not curative, but are aimed at managing symptoms through the 
reduction of inflammation. The mainstay of therapy remains use of topical corticosteroids which can be 
delivered in various vehicles including gels and solutions1,5,6. A response to treatment with midpotency 
corticosteroids (e.g., triamcinolone acetonide 0.1%), potent fluorinated corticosteroids (e.g., 
fluocinolone acetonide 0.1% and fluocinonide 0.05%), and super- potent halogenated corticosteroids 
(e.g., clobetasol propionate 0.05%), has been reported in 30–100% of treated individuals7-12.  
One of the challenges in applying topical corticosteroids is the lack of adherence to the oral mucosa for 
an adequate duration for the medication to be locally absorbed. Numerous obstacles are encountered 
when a topical steroid is prescribed to treat OLP, including the presence of saliva, changes in taste, 
poor tissue penetration, enzymatic degradation, and the need for frequent dosing, all of which may 
negatively affect patient compliance13. One approach to develop more efficacious and safer therapies 
could be inflammation-targeting drug delivery to achieve high drug concentrations locally at the site of 
inflammation with minimal exposure of healthy or distant tissues.  
Use of topical steroids for oral lichen planus may lead to the development of secondary candidiasis in 
11-47% of patients, which requires antifungal therapy10,14,15. Antifungal drug therapy, such as 
fluconazole has been showed to be an effective prophylaxis measure to prevent oral candidiasis 


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infection16-18. Although there are some reports of systemic absorption and adrenal suppression from 
super-potent topical steroids in the treatment of chronic dermatologic disorders, adrenal suppression is 
uncommon even in long-term oral application of topical corticosteroids such as fluocinonide and 
clobetasol19-21. Systemic absorption from topical steroids has been reported and it is thought that 
absorption of small amounts through the oral mucosa can take place, but clinical experience and 
laboratory studies have shown this not to be of clinical significance in almost all cases22. Gonzalez-
Moles et al. reported two cases of moon facies (2/30) and two cases of hirsutism (2/30) secondary to 
long-term use (> 4 weeks) of clobetasol 0.05% solution for the treatment of oral erosive lesions. All 
cases resolved upon discontinuation of the medication and/or reduction in frequency of administration.  
Other side effects reported include nausea, mucosal atrophy, xerostomia, halitosis and delayed 
healing22,23.  
 
1.2 
Dexamethasone solution for oral lichen planus 
  
The efficacy of dexamethasone solution has been well described in several clinical trials. Rhodus et al. 
conducted a prospective controlled clinical trial to test the efficacy of dexamethasone solution in 13 
patients with oral erosive-ulcerative lichen planus24. Patients were instructed to rinse with 5 ml of 
dexamethasone 0.1% solution for 3 min and then to expectorate. Subjective symptomatology (pain 
and/or discomfort) was recorded on a 100 mm visual analogue scale (VAS) at first visit and at 6 week 
follow up. Patients experienced significant subjective improvement in symptoms following the 
dexamethasone treatment (VAS score pre-treatment: 6.7 ± 1.4; VAS score post-treatment: 2.3 ± 0.6).  
Hambly et al. conducted a single-blinded, cross-over pilot trial in nine patients to compare the efficacy 
of compounded dexamethasone solution for OLP25. Patients were instructed to rinse with 2 ml of 
dexamethasone 0.5 mg/2 mL compounded as a solution or one 0.5 mg tablet crushed and mixed with 
20 ml water three times per day for 3 weeks. All patients were instructed to rinse and hold the 
medication in their mouths for at least 2–3 minutes and then expectorate. Participants were evaluated at 
weeks 0, 3, 4, and 7. Symptoms were recorded with a 100-mm VAS; other assessments included the 
Treatment Satisfaction Questionnaire for Medication-9, comparisons of clinical photographs, and self-
assessment via patient daily diary entries to record compliance with each dose. Results showed the 
compounded dexamethasone solution to be more effective compared to 0.5 mg dexamethasone tablet 
crushed and mixed with 20 ml water in terms of compliance, patient-perceived faster onset of action, 
and improved symptom relief. 
 
1.3 
Mucolox™ 
 
Mucolox™ is a mucoadhesive polymer gel that can be used in pharmaceutical compounding for the 
management of diseases and conditions of the oral mucosa including oral mucositis, and mouth 
ulcers26,27. The safety and toxicological profile of Mucolox™ was evaluated in vitro and compared to 
Triton X- 100 (positive control) and distilled water (negative control), using a 3-dimensional model of 
the human oral mucosa. Results showed that Mucolox™ can bind to tissues six times longer than 
Triton X-100 (before 50% cell viability) therefore offering increased retention of medication at the site 
of action. Additionally, at 4.5 hours, the difference in percent cell viability between Mucolox™ and 
distilled water was not significant, making it a safe water-soluble mucosal delivery system.  
 


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1.4 
Significance 
There is a significant unmet need for optimized topical therapies for oral lichen planus. Topical steroid 
therapy is considered the first line of treatment for oral lichen planus with current treatment regimens 
requiring multiple application or rinses daily24,28,29. Although topical steroid therapy can be successful 
for most patients, the treatment schedule can be challenging to maintain. There is therefore 
considerable interest in developing new and more effective therapies that require less frequent 
applications. The possibility of having a treatment agent that is safe, easy to use, and cost effective, 
with potentially greater efficacy than the current standard of care available topical steroid solutions 
would be ideal. Using Mucolox™ as a vehicle to deliver topical dexamethasone to the oral mucosa has 
the potential to effectively prolong contact time between the medication and mucosa, leading to 
improved clinical outcomes due to the need for less frequent application. This technology would help 
to more efficiently achieve high drug concentrations locally. By possibly shortening the frequency and 
time of topical therapy and improving patient compliance, this may translate to greater therapeutic 
benefit.  
2. SPECIFIC AIMS 
 
The objective of this single center, 4-week, open label randomized, phase II study is to evaluate and 
characterize the tolerability and clinical effectiveness of dexamethasone 0.5mg/5ml solution in a 
mucoadhesive vehicle (Mucolox™) for the treatment of oral lichen planus. Completion of this study 
will establish important data on the efficacy of topical steroid therapy when combined with a carrier 
and delivery system such as Mucolox™. If significant benefits are noted, this approach could be 
applied to the management of other oral mucosal immune-mediated conditions, such as graft-versus-
host disease and mucous membrane pemphigoid. 
 
2.1 
Study Design 
 
This is a single center, 4-week, open label randomized, phase II study. Randomization will be 
conducted by the BWH Investigational Pharmacy. The study period will be four weeks, which based on 
previous studies should be an adequate period to assess significant clinical improvement. Subjects will 
be evaluated clinically at baseline before starting treatment and at the end of the four-week period, for a 
total of two visits. Subjects will be randomized to either compound dexamethasone in Mucolox™ or 
dexamethasone only. Subjects will return for evaluation after four weeks at which time the primary 
end-points will be assessed.  
 
2.2 
Primary Objective 
 
The primary objective of this study is to determine the clinical efficacy and tolerability of compound 
dexamethasone at 0.5 mg/5 mL in Mucolox™ for the treatment of oral lichen planus as measured by a 
reduction in oral symptoms between patients treated with commercial dexamethasone 0.5mg/5ml 
solution in Mucolox™ (group A) and patients treated with topical dexamethasone 0.5mg/5ml solution 
only (group B). 
 
2.3 
Secondary Objective 
 


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The secondary objectives are: 
• To determine the clinical improvement in clinician-reported outcome measures achieved by 
treatment with topical compound dexamethasone at 0.5 mg/5 mL in Mucolox™ compared to 
dexamethasone 0.5mg/5ml solution alone. 
• To assess the tolerability of oral lichen planus as measured with the chronic oral mucosal 
diseases quality of life questionnaire (COMDQLQ). 
 
2.4 
Hypothesis 
 
Dexamethasone solution (0.5mg/5ml) in Mucolox™ when swished for 5 minutes and expectorated 
three times daily, is well-tolerated and more effective than dexamethasone solution (0.5 mg/5 mL) 
alone in the management of oral lichen planus.  
 
2.5 
Specific Aim  
In order to evaluate the hypothesis, the specific aim for this study is: 
2.5.1 To compare the clinical outcome with a primary endpoint of reduction in oral symptoms 
between patients treated with dexamethasone 0.5mg/5ml solution in Mucolox™ (group A) and 
patients treated with topical dexamethasone 0.5mg/5ml solution only (group B). 
 
3. SUBJECT SELECTION 
 
3.1 
Eligibility Criteria 
 
3.1.1 Age 18 years and older.   
 
3.1.2 Patients with symptomatic oral lichen planus (worst VAS sensitivity score ≥ 7 over the last 
week). 
 
3.1.3 Ability to understand and the willingness to sign a written informed consent document. 
 
 
3.2 
Exclusion Criteria 
 
3.2.1 Patients already on topical or systemic steroids. 
 
3.2.2 Inability to comply with study instructions. 
 
3.2.3 Uncontrolled intercurrent illness or psychiatric illness/social situations that would limit 
compliance with study requirements. 
 
3.2.4 VAS sensitivity score < 7. 
 
3.2.5 Pregnant women. A urine pregnancy test will be performed for women of child bearing potential. 
 


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3.2.6  Allergy to fluconazole, clotrimazole or nystatin. 
 
 
3.3 
Inclusion of Women and Minorities 
 
Both men and women of all races and ethnic groups are eligible for this trial. 
 
 
4. SUBJECT ENROLLMENT 
 
The Division of Oral Medicine and Dentistry at Brigham and Women’s Hospital is a major referral 
center for oral medicine patients including patients with oral lichen planus. Clinical diagnosis of oral 
lichen planus will be made by the patient’s oral medicine specialist. Patients with a confirmed clinical 
diagnosis of oral lichen planus and with oral symptoms (defined as the worst VAS sensitivity score ≥7 
over the last week) will be eligible to participate in this study. Potentially eligible subjects will be 
screened by one of the investigators by asking patients to rate their worst oral pain score (0-10) over the 
previous week (see Appendix).  Those answering with at least a score of “7” (1/10) and that meet all 
other eligibility requirements (see section 3.0) will be eligible for enrollment.  All patients (and/or 
parents/guardians) will sign informed consent for participation. All patients will be offered the 
opportunity to take home the consent form, and call back if they wish to participate. 
 
4.1 
Procedures for obtaining informed consent 
 
All patients will sign an informed consent. Patients will be informed that they have equal chances of 
being assigned to one of two groups. Patients will be told what personal identifying information will 
need to be collected, and the privacy rights of study participants will be explained. The consenting 
physician will ensure that the patient is given full and adequate oral and written information about the 
nature, purpose, and possible risks and benefits of the study, with the clear understanding that 
participation is optional. Patients will be encouraged to ask questions and will have the opportunity to 
take the consent form with them and review before making a decision whether or not to participate. 
Patients will also be notified that they are free to discontinue participation in the study at any time. By 
signing the consent form, potential subjects agree to participate in the study. If consent is withdrawn 
during the study, no further measurements will be conducted.  
 
4.2 
Treatment assignment and randomization 
 
Randomization will be predetermined by a computer generated list and coordinated by the BWH 
Investigational Pharmacy. Patients will be assigned to one of two groups: 1) Arm A: commercial 
dexamethasone 0.5mg/5ml solution in Mucolox™ or 2) dexamethasone 0.5mg/5ml solution only 
(ARM B).   
 
 
5. STUDY PROCEDURES 
 
Potentially eligible subjects will be screened by one of the investigators by asking patients to rate their 
worst oral pain or pain score (0-10) over the previous week (see Appendix). Those answering with at 


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11 
 
least a score of “7” (1/10) and that meet all other eligibility requirements (see Section 3) will be eligible 
for enrollment. All patients will sign informed consent for study participation. Each study visit is 
anticipated to take approximately 45 minutes.  A urine pregnancy test will be performed for women of 
child bearing potential. 
 
There is no financial compensation for participating in this study. Treatment will be administered on an 
outpatient basis. Study medication will be prescribed by authorized study staff physicians and filled by 
the BWH Investigational Pharmacy at no expense for the patient. No investigational or commercial 
agents or therapies other than those described below may be administered with the intent to treat the 
participant’s oral lichen planus.  
 
Subjects will be evaluated clinically at baseline before starting treatment and at the end of the four-
week period, for a total of two visits. Comprehensive subjective and objective data will be collected 
(Appendix 3 & 4) and intraoral photographs will be obtained. Oral mucosal disease will be evaluated 
using both patient reported (questions/visual analog scales) and clinician assessed measures.   
 
Subjects will be prescribed compound dexamethasone 0.5mg/5ml solution in Mucolox™ (ARM A) or 
dexamethasone 0.5mg/5ml solution only (ARM B). All subjects will also receive a prescription for 
fluconazole 200 mg tablets once-a-week as prophylactic antifungal therapy. Any subjects that are 
already taking an antifungal oral medication at the time of the study enrollment will continue their 
prescribed medication and will not need to take the additional weekly fluconazole dose. Subjects will 
return for evaluation after four weeks at which time the study end-points will be assessed.  
 
Clotrimazole or Nystatin will be prescribed as an alternative antifungal drug in the event of fluconazole 
allergy.  
 
If there is worsening of oral lichen planus that requires initiation of new immunomodulatory 
medications (systemic or topical), patients will remain on treatment, but will be regarded as 
unevaluable for the primary endpoint. 
 
 
 
 
 
 
Day 1 
(baseline) 
Day 28 + 5 days 
(end of study) 
Clinical examination 
x 
x 
COMDQLQ 
x 
x 
Patient Reported 
Outcomes 
x 
x 
 
5.1.1 Clinician Reported Outcomes 
 
Mucosal disease will be measured using the REU scoring system for monitoring oral lichen planus30.  
Assessment requires only a light source and a dental mirror. This is a validated semiquantitative 
instrument for monitoring reticular/hyperkeratotic, erosive/erythematous, and ulcerative OLP lesions. 
The oral cavity is divided into ten sites: right buccal mucosa, left buccal mucosa, tongue dorsum, 
tongue ventrum, maxillary gingiva, mandibular gingiva, floor of mouth, hard palatal mucosa, soft 


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12 
 
palate and tonsil, and labial mucosa (upper and lower together). The severity of each type of lesion is 
scored according to the presence or absence of reticulations/hyperkeratosis (0 = absent; 1 = present), 
and the size of the erosions/erythema or ulcers (in cm2) (0 = no involvement; 1-3 in increasing area of 
involvement). Because each type of lesion causes different degrees of discomfort and pain, each type of 
lesion is weighted accordingly: reticular lesions are weighted 1 because this presentation tends to be 
asymptomatic; erosive/erythematous lesions are weighted 1.5 since they tend to cause some degree of 
discomfort; ulcerative lesions are weighted 2.0 since they tend to be the most painful. The scores for 
each site are then totaled.  
 
Intraoral photographs will be obtained at each visit.    
 
5.1.2 Chronic Oral Mucosal Diseases Quality of Life Questionnaire 
 
Patients will complete the 24-item Chronic Oral Mucosal Diseases Quality of Life Questionnaire 
(COMDQLQ) instrument at baseline and at the end of treatment31. This is a validated instrument that 
assesses various aspects of oral health and function that has been shown to perform well in patients 
with oral mucosal diseases, including oral lichen planus.  
 
5.1.3 Patient Reported Outcomes   
 
Subjective assessments of subjects’ oral symptoms will be obtained by using instruments from the NIH 
consensus documents for oral cGVHD, a condition that is very similar to oral lichen planus (see 
Appendix 2&4). These include report of mouth sensitivity at rest and sensitivity with stimulation (e.g. 
eating) on an eleven point scale. For the primary endpoint the worst sensitivity in the past week will be 
used. The clinician seeing the subject will complete these forms at each study visit. Patients will also 
report daily sensitivity on an eleven point scale (see Treatment diary). Tolerability and compliance will 
also be evaluated, and a patient reported global assessment of overall improvement will be evaluated at 
the four-week visit. 
 
5.2 
Treatment Regimen 
 
A four week supply of compound dexamethasone 0.5mg/5ml solution in Mucolox™ (Arm A) and 
dexamethasone 0.5mg/5ml solution (Arm B) will be dispensed. The treatment instructions will be to 
rinse and spit (NOT swallow) with one teaspoon (5 mL) of solution, three times a day for 5 minutes at 
a time, and not to eat or drink for 15 minutes afterwards.  Subjects will maintain a diary and record 
each dose, the length of time rinsing, and any adverse effects (e.g. transient burning). 
 
Regimen Description 
 
Agent 
Premedications; 
Precautions* 
Dose 
Route 
Schedule 
Cycle 
Length 
Arm A  
Dexamethasone 
in Mucolox™  
None 
 
5mL x 
0.1mg/mL = 
0.5 mg/rinse, 
3 times/day 
=1.5 mg  
Oral Rinse, 5 
mins. each time 
(do not swallow) 
 
 7 
days/week 
28 days  
(4 
weeks) 
One 
Cycle 
Only* 


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13 
 
 
 
 
Arm B 
(standard 
of care) 
Dexamethasone 
solution  
None 
5mL x 
0.1mg/mL = 
0.5 mg/rinse, 
3 times/day 
=1.5 mg 
 
Oral Rinse, 5 
mins. each time  
(do not swallow) 
 7 
days/week 
28 days  
(4 
weeks) 
One 
Cycle 
Only* 
 
*All subjects will be treated with antifungal dosing for prophylaxis (see section 5.5) 
  
 
 
5.3 
Study Agents Administration 
 
The Investigator or co-investigators will prescribe the study drug in the clinic via Epic and instruct the 
patient on study drug use. Both dexamethasone compound 0.5mg/5ml solution in Mucolox™ and 
dexamethasone solution will be dosed topically at a concentration of 0.5 mg/5mL. 
 
A four week supply will be dispensed. The treatment instructions will be to rinse and spit (NOT 
swallow) with one teaspoon (5 mL) of the solution, three times a day for 5 minutes at a time, and not to 
eat or drink for 15 minutes afterwards. Subjects will maintain a diary and record each dose, the length 
of time rinsing, and any adverse effects. 
 
5.4 
Drug Accountability  
 
Patients will be instructed to bring empty bottles of study drug at the final visit. Adherence with the 
prescribed study treatment regimen will be determined by reviewing the study diary and returned 
bottles.   
 
5.5 
General Concomitant Medication and Supportive Care Guidelines 
 
There will be no restrictions on concomitant medications or supportive care measures.  
All subjects will be treated with fluconazole 200 mg tablets once-a-week dosing for prophylaxis as 
topical steroid therapy may increase the risk of candidiasis. This is typically very effective at 
preventing candidiasis in susceptible patients, and since there are no interim clinical visits, this will 
minimize the risk of secondary infection, which if not detected early, could confound the results at the 
week 4 evaluation. A single weekly dose of fluconazole is not sufficient to have an impact on 
therapeutic levels of other medications. Fluconazole is a pregnancy category D drug. A such, pregnant 
women will be excluded from this study.    
Any subjects that are already taking a systemic antifungal medication at the time of study enrollment 
will continue their prescribed medication and will not need to take the additional weekly fluconazole 
dose. 
 
Clotrimazole or nystatin will be prescribed as an alternative antifungal drug in the event of fluconazole 


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14 
 
allergy. Clotrimazole 10 mg troche (lozenge) to be dissolved in the mouth four times a day for four 
weeks; or nystatin 100,000 unit/mL oral suspension to rinse for one minute, swish and spit, four times a 
day for four weeks.  
 
Possible side effects of fluconazole include nausea, vomiting and headache. Possible side effects of 
nystatin oral suspension include rash, diarrhea, nausea, oral irritation, vomiting. Possible side effects of 
clotrimazole troche (lozenge) include abnormal sensation in the mouth, loss of appetite, diarrhea, 
nausea, vomiting, abnormal liver function tests. 
 
 
5.6 
Criteria for Taking a Participant Off Protocol Therapy 
 
Subjects will be taken off protocol therapy for any of the following reasons: 
 
• If the principal investigator feels it is in their best interest  
 
• If the subject cannot tolerate the study rinses  
 
• Intercurrent illness that prevents further administration of treatment 
 
• Unacceptable adverse event(s) 
 
• Participant demonstrates an inability or unwillingness to comply with the oral medication 
regimen and/or documentation requirements 
 
• Participant decides to withdraw from the protocol therapy 
 
• General or specific changes in the participant's condition render the participant unacceptable for 
further treatment in the judgment of the treating investigator 
 
 
Participants will be removed from the protocol therapy when any of these criteria apply. The reason for 
removal from protocol therapy, and the date the participant was removed, must be documented in the 
case report form (CRF). Alternative care options will be discussed with the participant.  
 
Subjects may be removed from the study in favor of clinical care should their symptoms worsen or 
should they have an adverse reaction to the study drug. 
 
In the event of unusual or life-threatening complications, treating investigators must immediately notify 
the Overall PI Alessandro Villa, DDS, PhD, MPH at 617-732-5517 (p36572). 
  
5.7 
Duration of Follow Up 
 
There is no follow-up period after completing the four-week treatment period. 
 


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15 
 
5.8 
Criteria for Taking a Participant Off Study 
 
Participants will be removed from study when any of the following criteria apply: 
• Lost to follow-up 
• Withdrawal of consent for data submission 
• Death 
 
The reason for taking a participant off study, and the date the participant was removed, will be 
documented in the case report form (CRF). 
  
 
6. BIOSTATISTICAL ANALYSIS 
 
6.1 
Study design/Endpoints 
 
The primary objective of this open label randomized phase II study is to evaluate the efficacy and 
tolerability of topical compound dexamethasone solution in a Mucolox™ for the treatment of oral 
lichen planus. The primary endpoint will be a change from pre to post treatment in each subject’s 
subjective sensitivity score (0-10) evaluated at baseline and 4 weeks post treatment for each arm. We 
will also compare changes of the daily subject’s subjective sensitivity score (0-10) over time for each 
arm. 
 
The secondary objectives include the evaluation of objective data using the REU scoring system and 
the oral health related quality of life using the 24-item Chronic Oral Mucosal Diseases Quality of Life 
Questionnaire. For the analysis of secondary endpoints see section 6.5. 
  
 
 
6.2 
Sample Size, Accrual Rate and Study Duration 
 
The sample size was calculated based on the data by Rhodus et al., as to ensure at least 80% power and 
two-sided alpha of 0.05 in detecting a t difference of 2.0 points oral pain on an 11 point scale (VAS 0-
10) between Arm A and B24. The mean expected change for Arm A will be 6.5 points reduction with a 
standard deviation of 1.4 points. The mean expected change for Arm B will be 4.5 points reduction 
with a standard deviation of 1.4 points.   
 
A total of 12 patients per arm will be enrolled (total of 24 subjects). The proposed sample size includes 
a loss of follow up of 5%. Approximately 75 patients per year are diagnosed with oral lichen planus at 
the Division of Oral Medicine and Dentistry (BWH)32. We anticipate the accrual rate will be 
approximately 5 patients per month, thus the accrual goal will be achieved in approximately five 
months.    
 
 
6.3 
Interim Monitoring Plan 
 
Involvement in this study as a participating investigator implies acceptance of potential audits or 
inspections, including source data verification, by representatives designated by BWH or the BWH 


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16 
 
Overall Principal Investigator. The purpose of these audits or inspections is to examine study-related 
activities and documents to determine whether these activities were conducted and data were 
recorded, analyzed, and accurately reported in accordance with the protocol, institutional policy, 
Good Clinical Practice (GCP), and any applicable regulatory requirements.  
 
All data will be monitored for timeliness of submission, completeness, and adherence to protocol 
requirements. Monitoring will begin at the time of participant registration and will continue during 
protocol performance and completion 
 
6.3.1 Monitoring of Adverse Events 
 
All adverse events will be reviewed by the principal investigator or covering clinician. In accordance 
with PHRC guidelines, the PI or covering clinician will determine whether an adverse event was 
expected and/or related to study procedures. Study subjects will be asked to report any adverse events 
or concerns to study staff as they arise.  
 
6.3.2 Protocol Review and Amendments  
 
This protocol, the proposed informed consent and all forms of participant information related to the 
study (e.g., advertisements used to recruit participants) and any other necessary documents will be 
submitted, reviewed and approved by a properly constituted IRB governing each study location.  
 
Any changes made to the protocol will be submitted as amendments and will be approved by the 
IRB prior to implementation. Any changes in study conduct will be reported to the IRB. The BWH 
Overall Principal Investigator will disseminate protocol amendment information to all participating 
investigators.  
 
All decisions of the IRB concerning the conduct of the study will be made in writing. 
 
  
6.4 
Analysis of Primary Endpoints 
 
VAS scores will be summarized descriptively for each arm at pre-treatment, post-treatment, and pre-to-
post treatment change. The pre-to-post treatment change will be compared between the arms using 
either Student’s t-test or Mann–Whitney test, depending on the distribution of the data. 
 
6.5 
Analysis of Secondary Endpoints 
 
Oral health related quality of life will be assessed before and after topical therapy using the 24-item 
Chronic Oral Mucosal Diseases Quality of Life Questionnaire (COMDQLQ; see Appendix) instrument 
that measures subject's perceptions of the impact of oral conditions on their well-being. The 
COMDQLQ scores will be summarized descriptively for each arm at pre-treatment, post-treatment, and 
pre-to-post treatment change. The pre-to-post treatment change for each item and for all items 
combined will be assessed within each arm using either paired t test or Wilcoxon-signed-rank test 
depending on its distribution. In addition, each question will be dichotomized and classified as 
‘response’ if the answer is ‘not at all’, or ‘slightly’ to a question such as “how isolated do you feel as a 


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17 
 
result of this oral condition”, and pre-to-post treatment improvement will be assessed using McNemar’s 
test within each arm.   
 
In order to evaluate the clinical improvement in clinician-reported outcome measures for oral lichen 
planus, REU scores within the compound dexamethasone solution in Mucolox™ group (Arm A) will 
be compared to that in the dexamethasone solution only group (Arm B).  The REU scores will be 
summarized descriptively for each arm at pre-treatment, post-treatment, and pre-to-post treatment 
change. The pre-to-post treatment change will be compared between the arms using either student’s t-
test or Mann–Whitney test, depending on the distribution of the data. 
 
6.6 
Reporting and Exclusions 
 
Compliance will be monitored closely. Non-compliant patients who use the treatment fewer than 2 
times a day for more than 2 weeks (as determined by review of the treatment diary at the week 4 visit) 
will be considered unevaluable for the primary endpoint and replaced with new patients. These 
patients, however, will be included in the toxicity report. During the four-week period when patients 
are receiving study treatment, additions of new immunomodulatory medications (systemic or topical in 
the mouth) are allowed, but these patients will be regarded as unevaluable for the primary endpoint and 
will be replaced.   
 
6.6.1 Evaluation of Toxicity 
 
All participants will be evaluable for toxicity from the time of their first treatment. All toxicities will 
be monitored closely.   
 
Development of grade III-IV treatment related toxicity will be monitored closely.  If we observe 3 or 
more patients with grade III-IV treatment related toxicity, the study will be terminated.   
 
 
7. DOSING DELAYS/DOSE MODIFICATIONS 
 
All adverse events experienced by participants will be collected from the time of the first dose of study 
treatment, through the study and until the final study visit. Participants continuing to experience 
toxicity at the final study visit may be contacted for additional assessments until the toxicity has 
resolved or is deemed irreversible. 
 
There will be no dosing delays/modifications. 
 
8. RISKS AND DISCOMFORTS 
8.1 
Drug side effects and toxicities 
 
Potential side effects with dexamethasone 0.5mg/5mL solution used as an oral topical rinse include an 
increased risk of developing oropharyngeal candidiasis and systemic absorption. All patients will be 
prescribed antifungal prophylaxis while on study with either fluoconazole, clotrimazole or nystatin (see 


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18 
 
section 5.5).  All participants will be informed that they may withdraw from the study at any time. As a 
topically applied agent, dexamethasone therapy can be discontinued at any time without any 
complications. Any noted clinical response will be expected to be reversed in such situations.  
 
Prolonged application of potent topical corticosteroids (e.g., > 50 g of clobetasol propionate or > 500 g 
of hydrocortisone per week) may lead to short-term hypothalamic-pituitary-adrenal axis alteration. In 
our study, in case of any systemic absorption, the maximum dose would be 10.5 mg/week  (5 mL x 0.1 
mg/mL = 0.5 mg/rinse, 3 times/day = 1.5 mg, 7 days/week = 10.5 g). We do not expect there to be 
significant systemic absorption, and therefore do not expect systemic side effects of dexamethasone 
solution when used as a swish and spit. Mucolox™ is a vehicle for drug delivery used in compounding 
medications with ingredients expected to be harmless (see section 11.1.1).   
 
9. POTENTIAL BENEFITS 
 
9.1 
Potential benefits for participating individuals 
 
All subjects will be assigned an active treatment. Thus, it is anticipated that the majority of participants 
may benefit from participation in this study. Additionally, most participants may benefit from the 
satisfaction in knowing that their involvement has a potential impact on the clinical care of future 
patients suffering from their condition. 
 
9.2 
Potential benefits to society 
 
The results of this study have the potential to impact clinical guidelines for the management of oral 
lichen planus.   
 
10. MONITORING AND QUALITY ASSURANCE 
 
10.1 
Safety monitoring 
 
The safety of participants and the overall study will be monitored continuously throughout the study 
period. At the 4 week visit, evaluating physicians will review the participants’ diaries for self-reported 
adverse events. Subjects will be instructed to contact their treating physician by phone or email or 
patient Gateway to report any unexpected side effects or side effects lasting more than 7 days, should 
they occur. Any necessary medical intervention will be handled by the treating physician. Data 
accuracy and protocol compliance will be monitored and assured by the Principal Investigator and 
toxicities will be reported promptly to the IRB (see below). 
 
10.2 
Adverse events 
 
Adverse event (AE) monitoring and reporting is a routine part of every clinical trial. This study follows 
Partners Health Care guidelines regarding the definitions and reporting policies of adverse events. The 
following list of reported and/or potential AEs (Section 10.3) and the characteristics of an Expected 
Toxicity (Section 10.4) will determine whether the event requires expedited reporting in addition to 
routine reporting. 


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19 
 
 
10.3 
Reported and/or potential AE 
 
AE include development of secondary oral candidiasis and short-term hypothalamic-pituitary-adrenal 
axis alteration (e.g., Cushingoid symptoms), although rare. 
  
10.4 
Expected Toxicities 
 
A list of the adverse events and potential risks associated with the agents administered in this study 
appear above and will determine whether dose delays and modifications will be made or whether the 
event requires expedited reporting in addition to routine reporting. 
 
Some expected toxicities include: transient burning, and the development of secondary oral candidiasis 
(which will be minimized with prophylactic antifungal drug therapy). 
 
Prolonged application of potent topical corticosteroids (e.g., > 50 g of clobetasol propionate or > 500 g 
of hydrocortisone per week) may lead to short-term hypothalamic-pituitary-adrenal axis alteration33. In 
our study, in case of any systemic absorption, the maximum dose would be 10.5 mg/week  (5 mL x 0.1 
mg/mL = 0.5 mg/rinse, 3 times/day = 1.5 mg, 7 days/week = 10.5 g). We do not expect there to be 
significant systemic absorption, and therefore do not expect systemic side effects of Mucolox™ and/or 
dexamethasone solution when used as a swish and spit.      
 
 
10.5 
Adverse Event Characteristics 
 
• CTCAE term (AE description) and grade:  The descriptions and grading scales found in the 
revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be 
utilized for AE reporting.  All appropriate treatment areas should have access to a copy of the 
CTCAE version 4.0.  A copy of the CTCAE version 4.0 can be downloaded from the CTEP 
web site http://ctep.cancer.gov/protocolDevelopment/electronic_applications/ctc.htm. 
 
• Attribution of the AE: 
- 
Definite – The AE is clearly related to the study treatment. 
- 
Probable – The AE is likely related to the study treatment. 
- 
Possible – The AE may be related to the study treatment. 
- 
Unlikely – The AE is doubtfully related to the study treatment. 
- 
Unrelated – The AE is clearly NOT related to the study treatment. 
 
10.6 
Expedited Adverse Event Reporting  
 
10.6.1 Investigators will report to the Overall PI any serious adverse event (SAE) that occurs after the 
initial dose of study treatment, during treatment, or within 30 days of the last dose of treatment 
on the local institutional SAE form.  
 
10.7 
Expedited Reporting to the Food and Drug Administration (FDA) 
 


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20 
 
The Overall PI, as study sponsor, will be responsible for all communications with the FDA. The 
Overall PI will report to the FDA, regardless of the site of occurrence, any serious adverse event that 
meets the FDA’s criteria for expedited reporting following the reporting requirements and timelines set 
by the FDA. 
 
10.8 
Routine Adverse Event Reporting 
 
All Adverse Events will be reported in routine study data submissions to the Overall PI on the toxicity 
case report forms. AEs reported through expedited processes (e.g., reported to the IRB, FDA, etc.) 
will also be reported in routine study data submissions. 
 
11. PHARMACEUTICAL INFORMATION 
 
A list of the adverse events and potential risks associated with the investigational agent administered in 
this study can be found in Section 10. 
 
11.1 
Mucolox™ 
 
11.1.1 Description 
 
Mucolox™ (PCCA, Houston, TX 77099) is a mucoadhesive polymer designed to improve 
mucoadhesion and prolong retention of medications at application sites within the oral mucosa. The 
composition of Mucolox™ is outlined in the table below. 
 
Ingredient Name  
INCI Name/Technical Name  Classification  
FDA 
Reference  
Potassium Sorbate NF  
Potassium Sorbate  
Subpart D – Chemical Preservatives  
21 CFR 
582.3640  
Sodium Benzoate NF  
Sodium Benzoate  
Subpart D – Chemical Preservatives  
21 CFR 
582.3733  
Glycerin USP (Natural)  
Glycerin  
Subpart B – General Purpose Food Additive 21 CFR 
582.1320  
Edetate Disodium USP 
Dihydrate  
Disodium EDTA  
FDA has no questions regarding intended 
use.  
GRN000363  
Pullulan  
Pullulan  
FDA has no questions regarding intended 
use in certain foods.  
GRN000099  
Tamarindus Indica Seed 
Polysaccharide  
Tamarindus Indica Seed 
Polysaccharide  
FDA has no questions regarding intended 
use.  
GRN000503  
Isomalt  
Isomalt  
Self-Determined GRAS (1996)  
GRN6G0321  
Simethicone USP  
Simethicone  
 
 
Sodium Hyaluronate  
Sodium Hyaluronate  
 
 
Poloxamer 407  
Poloxamer 407  
 
 
Zea Mays (Corn) Starch  
Zea Mays (Corn) Starch  
 
 
Carbomer  
Carbomer  
 
 
Water  
 
 
 
 


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21 
 
 
11.1.2 Compatibility 
 
N/A 
 
11.1.3 Handling 
 
There are no special handling requirements. 
 
11.1.4 Availability 
 
Compound dexamethasone solution in Mucolox™ will be dosed at a concentration of 0.5 mg/5mL and 
dispensed through the  BWH Investigational Pharmacy. The prescription for the patients in ARM B 
(dexamethasone) will also be dispensed through the BWH Investigational Pharmacy. 
 
11.1.5 Preparation 
 
Study subjects will be given a kit with the following, including detailed written instructions (see 
Appendix 1): 
 
ARM A 
• A four week supply of compound dexamethasone solution in Mucolox™ will be dispensed, with 
extra for error and spillage, which will be rounded to  480 mL bottle at a concentration of 0.5 
mg/5mL. 
• Small dosing cups 
 
  
11.1.6 Administration 
 
Subjects will be instructed to rinse with 5 ml of compound dexamethasone solution in Mucolox™ (0.5 
mg/5ml; ARM A) for 5 minutes. After 5 minutes, subjects will be instructed to expectorate and to not 
eat or drink for 15 minutes after. The rinses will be completed three times per day, equally spaced 
during awake hours. If a study rinse is missed, subjects will be asked to attempt to make up for the 
missed rinse later the same day, to maintain four rinses per day. If necessary, for example, two 
consecutive 5-minute rinses can be completed at night before bed. This does not pose any known safety 
risk.  
 
11.1.7 Ordering 
 
Study rinses will be ordered through the BWH Investigational Pharmacy via EPIC. 
 
11.1.8 Accountability 
 
A study diary will be given to the subject where they will be asked to check off each rinse completed, 
with three check boxes per day on study (Appendix 2). 
 


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22 
 
11.1.9  Destruction and Return 
 
Patients should not continue to use any unused study supply once they have completed the study. It 
should be returned to the investigator and will be sent to pharmacy for destruction. 
 
11.2 
Dexamethasone solution 
 
11.2.1 Description 
 
Dexamethasone (NDC 00054-3177-63; Roxane Laboratories, Columbus, OH 43216-6532) 
is a synthetic adrenocortical steroid commercially available as a 0.5mg/5mL solution. It is stable in air 
and practically insoluble in water.  
• The molecular weight is 392.47.  
• The molecular formula is C22H29FO5  
• The structural formula is:  
 
 
Each 5 mL of oral solution contains 0.5 mg of dexamethasone and citric acid, disodium edetate, 
flavoring, glycerin, methylparaben, propylene glycol, propylparaben, sorbitol and water. 
 
11.2.2 Storage and Stability 
 
Dexamethasone solution is stable at room temperature for 30 days after opening per package insert. 
 
 
11.2.3 Compatibility 
 
N/A 
 
11.2.4 Handling 
 
There are no special handling requirements. 
 
11.2.5 Availability 
 
Dexamethasone 0.5mg/5mL solution will be dispensed through the BWH Investigational Pharmacy.  
 
11.2.6 Preparation 
 
Study subjects will be given a kit with the following, including detailed written instructions (see 
Appendix 1): 
 


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23 
 
ARM B 
• A four week supply of dexamethasone solution will be dispensed, with extra for error and spillage, 
which will be rounded to 480 mL bottle at a concentration of 0.5 mg/5mL. 
• Small dosing cups 
 
  
11.2.7 Administration 
 
Subjects will be instructed to rinse with 5 ml of the prescribed dexamethasone 0.5mg/5ml solution 
(ARM B) for 5 minutes. After 5 minutes, subjects will be instructed to expectorate and to not eat or 
drink for 15 minutes after.  The rinses will be completed three times per day, equally spaced during 
awake hours.  If a study rinse is missed, subjects will be asked to attempt to make up for the missed 
rinse later the same day, to maintain four rinses per day. If necessary, for example, two consecutive 5 
minute rinses can be completed at night before bed. This does not pose any known safety risk.  
 
11.2.8 Ordering 
 
Dexamethasone solution will be ordered through the BWH Investigational Pharmacy via EPIC. 
 
11.2.9 Accountability 
 
A study diary will be given to the subject where they will be asked to check off each rinse completed, 
with three check boxes per day on study (Appendix 2). 
 
11.2.10 
 Destruction and Return 
 
Patients should not continue to use any unused study supply once they have completed the study. It 
should be returned to the investigator for destruction. 
 
 
12. DATA REPORTING / REGULATORY REQUIREMENTS 
 
Adverse event lists, guidelines, and instructions for AE reporting can be found in Section 10.0  
 
 
12.1 
Monitoring and quality assurance 
 
Safety monitoring 
The safety of participants and the overall study will be monitored continuously throughout the study 
period. Evaluating physicians will query participants for any adverse events and review self-reported 
adverse events. Subjects will be instructed to contact their treating physician by phone or email to 
report any unexpected side effects or side effects lasting more than 7 days, should they occur. Any 
necessary medical intervention will be handled by the treating physician. Data accuracy and protocol 
compliance will be monitored and assured by the Principal Investigator and toxicities will be reported 
promptly to the IRB (see above).   


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24 
 
 
13. PUBLICATION PLAN 
 
The principal investigator holds the primary responsibility for publication of the study results.  The 
results will be presented in abstract form at a national/international and/or oral medicine conference 
and will be published in a peer reviewed journal.  
 


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25 
 
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pathology, oral radiology, and endodontics. 2011;111(5):578-586, 586 e571-572. 
32. 
Villa A, Stock S, Aboalela A, et al. Oral Medicine referrals at a hospital-based practice in the 
United States. Oral Surg Oral Med Oral Pathol Oral Radiol 2015;In press. 
33. 
Mooney E, Rademaker M, Dailey R, et al. Adverse effects of topical corticosteroids in 
paediatric eczema: Australasian consensus statement. Australas J Dermatol. 2015;56(4):241-


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27 
 
251. 
 
 
 
 


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28 
 
 
APPENDICES 
Appendix 1: Patient instructions 
Appendix 2: Treatment diary  
Appendix 3: Visit #1 CRF 
Appendix 4: Visit #2 CRF 
 
Appendix 5:  COMDQLQ 
 


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29 
 
INSTRUCTIONS FOR PREPARING & USING YOUR 
TOPICAL SOLUTION 
 
 
WHAT YOU WILL RECEIVE 
 
• One 480 mL bottle of concentrated solution (5 mg/5 mL) 
 
USING YOUR SOLUTION 
  
You will be rinsing with your solution 3 times per day.    
 
1. Gently shake the bottle of solution. 
2. Pour 5 mL of solution into the dosing cups provided. 
3. Swish the solution in your mouth for 5 minutes, then spit – DO NOT SWALLOW! 
4. Do not eat or drink for 15 minutes after rinsing.   
 
 


 
Treatment Diary  
 
Week 
Day/Date 
Rinse #1 (check/fill) 
Rinse #2 (check/fill) 
Rinse #3 (check/fill) 
From a 
scale 0-10, 
how would 
you rate 
your mouth 
sensitivity 
today?  
Comments 
5 min 
Other* 
5 min 
Other* 
5 min 
Other* 
1 
1 
 
 
 
 
 
 
 
 
 
2 
 
 
 
 
 
 
 
 
 
3 
 
 
 
 
 
 
 
 
 
4 
 
 
 
 
 
 
 
 
 
5 
 
 
 
 
 
 
 
 
 
6 
 
 
 
 
 
 
 
 
 
7 
 
 
 
 
 
 
 
 
 
2 
8 
 
 
 
 
 
 
 
 
 
9 
 
 
 
 
 
 
 
 
 
10 
 
 
 
 
 
 
 
 
 
11 
 
 
 
 
 
 
 
 
 
12 
 
 
 
 
 
 
 
 
 


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31 
 
13 
 
 
 
 
 
 
 
 
 
14 
 
 
 
 
 
 
 
 
 
 
Week 
Day/Date 
Rinse #1 (check/fill) 
Rinse #2 (check/fill) 
Rinse #3 (check/fill) 
From a 
scale 0-10, 
how would 
you rate 
your mouth 
sensitivity 
today? 
Comments 
5 min 
Other* 
5 min 
Other* 
5 min 
Other* 
3 
15 
 
 
 
 
 
 
 
 
 
16 
 
 
 
 
 
 
 
 
 
17 
 
 
 
 
 
 
 
 
 
 
18 
 
 
 
 
 
 
 
 
 
19 
 
 
 
 
 
 
 
 
 
20 
 
 
 
 
 
 
 
 
 
21 
 
 
 
 
 
 
 
 
 
4 
22 
 
 
 
 
 
 
 
 
 
23 
 
 
 
 
 
 
 
 
 
24 
 
 
 
 
 
 
 
 
 


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32 
 
25 
 
 
 
 
 
 
 
 
 
26 
 
 
 
 
 
 
 
 
 
27 
 
 
 
 
 
 
 
 
 
28 
 
 
 
 
 
 
 
 
 


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33 
 
DEXAMETHASONE RINSE AND MUCOLOX™ FOR THE TREATMENT OF ORAL 
LICHEN PLANUS  
 
VISIT #1 
 
 
Subject Name  
 
 
 
 
 
 
 
 
 
 
Medical Record Number 
 
 
 
 
 
 
Date of Visit  
 
 
 
 
 
 
 
 
SUBJECTIVE DATA (Over last two weeks unless specified) 
 
MUCOSAL PATHOLOGY 
 
1. Does your mouth hurt now? 
 
 
 
 
 
 
 
Y/N 
 
2. Does it hurt to swallow food or drink?  
 
 
 
 
 
Y/N 
 
3. How would you rate your worst mouth sensitivity over the last week?  
 
No sensitivity  
 
 
 
 
 
Worst sensitivity 
 


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34 
 
 
4. Do you avoid any foods because they make your mouth hurt?   
  
 
 
 
0 = 
Not at all 
1 = 
Slightly 
2 = 
Moderately 
3 = 
Quite a bit 
4 = 
Extremely 
 
If “yes”, what types of foods? 
 
a. Spicy foods 
 
 
 
 
b. Acidic foods  
 
 
 
c. Salty foods 
 
 
 
 
d. Hard/crunchy/crusty foods 
 
 
e. Other: 
 
5. Do you feel that your oral intake (what you eat) is limited by your oral condition? 
Y/N 
 
Not limited at all 
 
 
    
               Severely limited 
 
 
Score: _____ 
 
If “Yes”, how limited do you feel it is? 
 
a. Partial limitation 
 
 
 
b. Severe limitation 
 
 
 
 
6. Have you been bothered by ulcers in the mouth?  
 
0 = 
Not at all 
1 = 
Slightly 
2 = 
Moderately 
3 = 
Quite a bit 
4 = 
Extremely 


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35 
 
  
OBJECTIVE DATA (REU score) 
 
 
(R: 0 = not present, 1 = present; E & U: 0 = not present, 1 = <1cm2, 2 = 1 - 3cm2, 3 = >3cm2) 
Total Score: 
Total Weighted Score: 
 
 
PHOTOGRAPHS TAKEN  (circle):  YES    NO 
 
 
 
 


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36 
 
 DEXAMETHASONE RINSE AND MUCOLOX™ FOR THE TREATMENT OF ORAL 
LICHEN PLANUS  
 
 
VISIT #2 
 
 
Subject Name  
 
 
 
 
 
 
 
 
 
 
Medical Record Number 
 
 
 
 
 
 
Date of Visit  
 
 
 
 
 
 
 
 
SUBJECTIVE DATA (Over last one week unless specified) 
 
MUCOSAL PATHOLOGY 
 
1. Does your mouth hurt now? 
 
 
 
 
 
 
 
Y/N 
 
2. Does it hurt to swallow food or drink?  
 
 
 
 
 
Y/N 
 
3. How would you rate your worst mouth sensitivity over the last week?  
 
No sensitivity 
 
 
 
 
 
 
Worst sensitivity 
 
4. Do you avoid any foods because they make your mouth hurt?   
  
 
 
 
0 = 
Not at all 
1 = 
Slightly 
2 = 
Moderately 
3 = 
Quite a bit 
4 = 
Extremely 
 
If “yes”, what types of foods? 
 
a. 
Spicy foods 
 
 
 
 
b. 
Acidic foods 
 
 
 
 
c. 
Salty foods 
 
 
 
 


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37 
 
d. 
Hard/crunchy/crusty foods 
 
 
e. 
Other: 
  
 
5. Do you feel that your oral intake (what you eat) is limited by your oral condition? 
Y/N 
 
Not limited at all 
 
 
    
        Severely limited 
 
 
 
 
 
Score: _____ 
 
If “Yes”, how limited do you feel it is? 
 
a. 
Partial limitation  
 
 
b. 
Severe limitation  
 
 
 
6. Have you been bothered by ulcers in the mouth?   
 
0 = 
Not at all 
1 = 
Slightly 
2 = 
Moderately 
3 = 
Quite a bit 
4 = 
Extremely 
 
 
 
7. Rate your level of comfort while rinsing with the topical therapy: 
 
Not uncomfortable at all 
 
 
               Very uncomfortable 
 
 
Score: _____ 
 


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38 
 
 
8. Rate the taste of the topical treatment: 
 
 Very good taste 
 
 
 
Very bad taste 
 
 
Score: _____ 
 
9. Rate how your mouth feels since starting topical therapy: 
 
Mouth feels much better 
                                     Mouth feels much worse 
 
 
Score: _____ 
 
10. Overall, since beginning topical therapy, my mouth is: (Check one) 
 
 Very much better  
 
 
 Moderately better 
 A little better  
 About the same 
 A little worse  
 Moderately worse 
 Very much worse 
 
 


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OBJECTIVE DATA 
 
 
 
 
(R: 0 = not present, 1 = present; E & U: 0 = not present, 1 = <1cm2, 2 = 1 - 3cm2, 3 = >3cm2) 
Total Score: 
Total Weighted Score: 
 
 
PHOTOGRAPHS TAKEN (circle):  YES    NO


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40 
 
COMDQLQ 
 
Not at all (0) 
Slightly (1) 
Moderately (2) 
Considerably (3) 
Extremely (4) 
Pain and functional limitation  
 
 
 
 
 
How much do certain types of 
food/drink cause you discomfort 
(spicy food, acidic food)?  
 
 
 
 
 
How much does your oral 
condition cause you to limit the 
types of food/ drinks you 
consume?  
 
 
 
 
 
How much do certain food 
textures cause you discomfort 
(rough food, crusty food)?  
 
 
 
 
 
How much does your oral 
condition cause you to limit the 
textures of the food you consume?  
 
 
 
 
 
How much does the temperature 
of certain foods/drinks cause you 
discomfort?  
 
 
 
 
 
How much does you oral 
condition cause you to limit the 
temperature of the foods/ drinks 
you consume?  
 
 
 
 
 
How much does your oral 
condition lead to discomfort when 
carrying out your daily oral 
hygiene routine (brushing, 
flossing, mouthwash usage)?  
 
 
 
 
 
How much does your oral 
condition cause you to limit your 
daily oral hygiene routine 
(brushing, flossing, mouthwash 
usage)?  
 
 
 
 
 
How much does your oral 
condition lead to discomfort when 
wearing a denture (false teeth)?  
 
 
 
 
 
Medication and treatment  
 
 
 
 
 
How much do you feel you need 
medication to help you with 
activities of daily life (talking, 
eating, etc.)?  
 
 
 
 
 
How satisfied are you with the 
medication being used to treat 
your oral condition?  
 
 
 
 
 
How concerned are you about the 
possible side effects of the 
medications used to treat your 
oral condition?  
 
 
 
 
 
How much does it frustrate you 
 
 
 
 
 


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41 
 
that there is no single standard 
medication to be used in your oral 
condition?  
How much does the use of the 
medication limit you in your 
everyday life (routine/the way 
you apply or take your 
medications)?  
 
 
 
 
 
How much does it bother you that 
there is no cure for your oral 
condition?  
 
 
 
 
 
Social and emotional  
 
 
 
 
 
How much does your oral 
condition you down?  
 
 
 
 
 
How much does your oral 
condition cause you anxiety?  
 
 
 
 
 
How much does your oral 
condition cause you stress?  
 
 
 
 
 
How much does the 
unpredictability your oral 
condition bother you?  
 
 
 
 
 
How much does your oral 
condition cause you to worry 
about the future (spread of the 
condition, possible cancer risk)?  
 
 
 
 
 
How much does your oral 
condition make you pessimistic 
about the future?  
 
 
 
 
 
How much does your oral 
condition disrupt social activities 
in your life (social gatherings, 
eating out parties)?  
 
 
 
 
 
Patient support  
 
 
 
 
 
How satisfactory do you consider 
the information available to you 
regarding your oral condition?  
 
 
 
 
 
How satisfied are you with the 
level of support and 
understanding shown to you by 
family regarding this oral 
condition?  
 
 
 
 
 
How satisfied are you with the 
level of support and 
understanding shown to you by 
friends/work colleagues regarding 
your oral condition?  
 
 
 
 
 
How isolated do you feel as a 
result of this oral condition?