Role of Neurogenic Inflammation and Topical 6% Gabapentin Therapy in Symptomatic Scarring Alopecia
Phase: EARLY_PHASE1
Conditions: Scarring Alopecia, Frontal Fibrosing Alopecia, Lichen Planopilaris, Central Centrifugal Cicatricial Alopecia, Central Centrifugal Scarring Alopecia
Sponsor: University of Minnesota

 
Scarring Alopecia-Gabapentin Study 
Version 5 | Approved 01.28.2016 
 
 
 
Title:    
 
 
      Role of Neurogenic Inflammation and Topical 6% Gabapentin  
 
 
 
 
      Therapy in Symptomatic Scarring Alopecia  
 
Protocol Number: 
1308M40801 
 
Study Drug: 
Gabapentin 6% Solution 
 
Principal Investigator: 
Maria Hordinsky, MD 
 
612-625-8625 
 
Study Team: 
Rehana Ahmed-Saucedo, MD, PhD 
 
Margareth Pierre-Louis, MD 
 
Elisabeth Hurliman, MD, PhD 
 
 
Trial Site: 
University of Minnesota, Department of Dermatology 
 
MMC 98 
 
Minneapolis, MN 55455 
 
Laboratory: 
University of Minnesota, Fairview Laboratory 
 
516 Delaware St.  
 
Minneapolis, MN 55455 
 
 
 
 
 
Masonic Clinical Research Unit 
 
MMC 405, 420 Delaware St. 
 
Minneapolis, MN 55455 
 
Version Date: 
January 12, 2016  
 
 
 
 
 
 
 
 
 
 
 
 
CONFIDENTIAL 
 
This is a University of Minnesota clinical research trial document that contains confidential 
information.  It is intended solely for the recipient clinical investigator(s) and must not be 
disclosed to any other party.  The material may be used only for evaluating or conducting 
clinical investigations; any other proposed use requires written consent from the University of 
Minnesota Department of Dermatology.   
 

 
 
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This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
Table of Contents 
STUDY SUMMARY ................................................................................................................................. 1 
1 
INTRODUCTION ............................................................................................................................... 2 
1.1 
BACKGROUND .............................................................................................................................. 2 
1.2 
INVESTIGATIONAL AGENT............................................................................................................... 2 
1.3 
PRECLINICAL DATA ....................................................................................................................... 3 
1.4 
CLINICAL DATA TO DATE ............................................................................................................... 3 
1.5 
DOSE RATIONALE AND RISK/BENEFITS ............................................................................................ 3 
2 
STUDY OBJECTIVES ....................................................................................................................... 4 
3 
STUDY DESIGN ............................................................................................................................... 4 
3.1 
GENERAL DESIGN ......................................................................................................................... 4 
3.2 
PRIMARY STUDY ENDPOINTS ......................................................................................................... 5 
3.3 
SECONDARY STUDY ENDPOINTS .................................................................................................... 6 
3.4 
PRIMARY SAFETY ENDPOINTS ........................................................................................................ 6 
4 
SUBJECT SELECTION AND WITHDRAWAL ................................................................................... 6 
4.1 
INCLUSION CRITERIA ..................................................................................................................... 6 
4.2 
EXCLUSION CRITERIA .................................................................................................................... 6 
4.3 
SUBJECT RECRUITMENT AND SCREENING ....................................................................................... 7 
4.4 
EARLY WITHDRAWAL OF SUBJECTS ................................................................................................ 7 
4.4.1 
When and How to Withdraw Subjects .................................................................................. 7 
4.4.2 
Data Collection and Follow-up for Withdrawn Subjects ........................................................ 8 
5 
STUDY DRUG................................................................................................................................... 8 
5.1 
DESCRIPTION ............................................................................................................................... 8 
5.2 
METHOD FOR ASSIGNING SUBJECTS TO TREATMENT GROUPS ........................................................... 9 
5.3 
PREPARATION AND ADMINISTRATION OF STUDY DRUG ...................................................................... 9 
5.4 
SUBJECT COMPLIANCE MONITORING .............................................................................................. 9 
5.5 
PRIOR AND CONCOMITANT THERAPY .............................................................................................. 9 
5.6 
PACKAGING ................................................................................................................................. 9 
5.7 
BLINDING OF STUDY DRUG ............................................................................................................ 9 
5.8 
RECEIVING, STORAGE, DISPENSING AND RETURN ............................................................................ 9 
5.8.1 
Receipt of Drug Supplies ................................................................................................... 10 
5.8.2 
Dispensing of Study Drug .................................................................................................. 10 
5.8.3 
Return or Destruction of Study Drug .................................................................................. 10 
6 
STUDY PROCEDURES .................................................................................................................. 10 
6.1 
VISIT 1 (SCREENING/BASELINE) .................................................................................................... 10 
6.2 VISIT 2 (DAY 0) ........................................................................................................................... 11 
6.3 VISIT 3 (WEEK 4) ........................................................................................................................ 11 
6.4 VISIT 4 (WEEK 8) ........................................................................................................................ 12 
6.5 VISIT 5 (WEEK 12) ...................................................................................................................... 12 
6.6 VISIT 6 (2 WEEK FOLLOW-UP) ....................................................................................................... 12 
7 
STATISTICAL PLAN ...................................................................................................................... 13 
7.1 
SAMPLE SIZE DETERMINATION ..................................................................................................... 13 
7.2 
STATISTICAL METHODS ............................................................................................................... 13 
7.3 
SUBJECT POPULATION(S) FOR ANALYSIS....................................................................................... 13 
8 
SAFETY AND ADVERSE EVENTS ................................................................................................. 13 
8.1 
DEFINITIONS .............................................................................................................................. 13 
8.2 
RECORDING OF ADVERSE EVENTS ............................................................................................... 15 
8.3 
REPORTING OF SERIOUS ADVERSE EVENTS .................................................................................. 15 
8.3.1 
Study Sponsor Notification by Investigator ......................................................................... 15 

 
 
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8.3.2 
IRB Notification by Investigator .......................................................................................... 16 
8.3.3 
FDA Notification by Sponsor .............................................................................................. 16 
8.4 
UNBLINDING PROCEDURES .......................................................................................................... 16 
8.5 
STOPPING RULES ....................................................................................................................... 16 
8.6 
MEDICAL MONITORING ................................................................................................................ 16 
9 
DATA HANDLING AND RECORD KEEPING ................................................................................. 17 
9.1 
CONFIDENTIALITY ....................................................................................................................... 17 
9.2 
SOURCE DOCUMENTS/CASE REPORT FORMS ................................................................................ 17 
9.3 
RECORDS RETENTION ................................................................................................................. 17 
10 
STUDY AUDITING, AND INSPECTING ........................................................................................ 17 
10.1 
AUDITING AND INSPECTING ........................................................................................................ 17 
11 
ETHICAL CONSIDERATIONS ...................................................................................................... 18 
12 
STUDY FINANCES ....................................................................................................................... 18 
12.1 
FUNDING SOURCE .................................................................................................................... 18 
12.2 RESEARCH RELATED INJURY ...................................................................................................... 18 
12.3 SUBJECT STIPENDS OR PAYMENTS ............................................................................................. 19 
12.4 
CONFLICT OF INTEREST ............................................................................................................. 19 
13 PUBLICATION PLAN .................................................................................................................... 19 
14 REFERENCES .............................................................................................................................. 19 
15 ATTACHMENTS ........................................................................................................................... 20 
APPENDIX A: DERMATOLOGY QUALITY OF LIFE INDEX (DLQI) ..................................................... 21 
APPENDIX B: SHORT FORM (36) HEALTH SURVEY (SF-36) ............................................................. 22 
APPENDIX C: ITCH VISUAL ANALOG SCALE (VAS) ......................................................................... 27 
APPENDIX D: PAIN VISUAL ANALOG SCALE (VAS) ......................................................................... 28 
APPENDIX E: LICHEN PLANOPILARIS ACTIVITY INDEX (LPPAI) ..................................................... 30 
APPENDIX F: ADVERSE EVENT REPORT FORM ............................................................................... 30 
 

 
 
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List of Abbreviations 
 
CGRP  
Calcitonin Gene-Related Peptide 
 
CFR  
Code of Federal Regulations 
 
CRF  
Case Report Form 
 
CTCAE 
Common Terminology Criteria for Adverse Events 
 
DLQI             Dermatology Quality of Life Index 
 
ELISA           Enzyme-linked Immunosorbent Assay 
 
FDA  
Food and Drug Administration 
 
GABA  
Gamma -Aminobutyric Acid 
 
GCP  
Good Clinical Practice 
 
ICH 
 
International Conference on Harmonization 
 
IDS   
Investigational Drug Services Pharmacy 
 
IRB 
 
Institutional Review Board 
 
H&E    
Hematoxylin and Eosin Stain 
 
LPP 
 
Lichen Planopilaris 
 
LPPAI  
Lichen Planopilaris Activity Index 
 
PSA  
Primary Scarring Alopecia 
 
SF-36  
Short Form (36) Health Survey 
 
UPIRTSO 
Unanticipated Problems Involving Risk to Subjects or Others 
 
VAS  
Visual Analog Scale 
 
 

 
 
Page 1 
 
Scarring Alopecia-Gabapentin Study 
Version 5 | Approved 01.28.2016 
Study Summary 
 
Title 
Role of Neurogenic Inflammation and Topical 6% Gabapentin Therapy 
in Symptomatic Scarring Alopecia  
Short Title 
Scarring alopecia – Gabapentin study 
Methodology 
Cohort study 
Study Duration 
16 weeks for each subject 
Study Center(s) 
Single-center 
Objectives 
To determine the role of neurogenic inflammation in scarring alopecia 
and to assess the safety and efficacy of topical gabapentin in the 
management of symptomatic scarring alopecia. 
Number of Subjects 
10 (ten) 
Diagnosis and Main 
Inclusion Criteria 
Biopsy-proven scarring alopecia; Men and women greater than 18 
years of age. Their scarring alopecia should be symptomatic with 
symptoms including burning, tingling, itch, pain in addition to hair loss. 
The subject should have no history of use of neuromodulatory agents 
two months prior to study enrollment.  
Study Product, 
Dose, Route, 
Regimen 
Gabapentin 6% solution 
The maximum daily dose of topical gabapentin will be 2 ml with the 
estimation that no more than one-third of a subject’s scalp is affected.  
The drug will be administered topically by the subject to symptomatic 
areas of intact skin two times daily for 12 weeks. Based on its half-life 
of 8 hours when taken orally, gabapentin will be administered two 
times a day and the dose will not exceed the amount of topical agent 
used on average to treat the entire scalp in everyday clinical practice 
(1 mL twice daily, 60 mL per month). 
Duration of 
administration 
The drug will be topically applied by the subject two times daily for 12 
weeks. 
Reference therapy 
This is a pilot study and there is no reference therapy against which 
topical 6% gabapentin will be compared. 
Statistical 
Methodology 
An intent-to-treat analysis will be performed in all subjects with 
symptoms comparing the last visit to baseline.  
 

 
 
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1 
Introduction 
This document is a protocol for a human research study. This study is to be conducted 
according to US and international standards of Good Clinical Practice (FDA Title 21 part 
312 and International Conference on Harmonization guidelines), applicable government 
regulations and Institutional research policies and procedures.  
 
1.1 
Background 
Primary scarring alopecias (PSAs) are poorly understood dermatologic disorders that 
result in permanent hair loss (1). PSAs are classified based on the type of inflammatory 
infiltrate recognized histologically on biopsy: lymphocytic, neutrophilic, or mixed.  As 
painful hair loss disorders, scarring alopecias have yet to be studied in a manner that 
explores the disease burden on the individual and how to more effectively manage 
debilitating symptoms.  Most of the scarring alopecias involve a painful course, with 
individuals reporting scalp pain, burning, itching, or tingling/crawling sensations that can 
ultimately impact physical and psychological health (1). It is well-documented that 
individuals with chronic pain are more susceptible to depression and other mood 
disorders (2). Thus, a better understanding of the pain process in scarring alopecia 
would benefit patients and health care providers tremendously as scientists research to 
discover the origin and pathogenesis of these inflammatory and irreversible conditions. 
 
CGRP (calcitonin gene-related peptide) has been proposed to play a role in pain 
transmission and inflammation (3, 4).  While originally found to be elevated in migraines, 
two patients with lichen planopilaris (LPP) at the University of Minnesota were found to 
have elevated amounts of CGRP via immunohistochemical staining and confocal 
microscopic examination of scalp biopsy specimens. Seven patients, three with frontal 
fibrosing alopecia and four with LPP, were also found to have decreased expression of 
epidermal nerves as well as CRGP expression.  This suggests that neurogenic 
inflammation may contribute to the disease state of scarring alopecia. Investigating this 
relationship between CGRP and scalp symptoms such as pain in the setting of scarring 
alopecia may lead to a long awaited breakthrough in disease pathogenesis and 
management. Agents that reduce the release of neuropeptides, such as gabapentin, 
may serve as potential treatment in scarring alopecia. Recent data from pilot 
experiments at the University of Minnesota using normal human tissue stimulated by 
gabapentin reflected a reduction in CGRP compared to levels of CGRP in control 
unstimulated tissue, indicating that CGRP may realistically be an appropriate 
therapeutic target in scarring alopecia. 
 
1.2 
Investigational Agent 
Gabapentin is a GABA analogue originally introduced in 1994 for the treatment of 
epilepsy by Warner-Lambert (today Pfizer). It is currently widely used to relieve pain, 
especially neuropathic pain syndromes associated with diabetic neuropathy, vulvodynia, 
postherpetic neuralgia and other allodynia (5, 6). While gabapentin’s mechanism of 
action is not completely understood, it is thought to work through its effects on voltage-
gated calcium ion channels in the central nervous system once it crosses the blood-
brain barrier (7). Its neuromodulatory effects also impact the peripheral nervous system.  
 

 
 
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1.3 
Preclinical Data 
Currently, the management of symptomatic scarring alopecia includes intralesional 
triamcinolone injections alone or with topical steroid solutions, minoxidil, oral antibiotics, 
hydroxychloroquine or immunosuppressive medications such as prednisone.  Since the 
cause of scarring alopecia is unknown, the various modes of treatment have focused on 
targeting the inflammation associated with the disease process that is reflected 
histologically. No one treatment has been found successful in all conditions nor is there 
a cure. Individuals with end-stage, non-symptomatic disease may be candidates for hair 
transplantation.   
 
1.4 
Clinical Data to Date 
There has been no study of topical neurogenic agents, such as gabapentin, to treat 
scarring alopecia. However topical gabapentin has been safely used in other conditions 
associated with chronic pain, burning, irritation, itch, or tingling, such as vulvodynia (5).  
 
Interestingly, gabapentin has been shown to be effective against migraine headaches, a 
condition associated with high levels of CGRP (6).  Gabapentin has also been shown to 
be effective in managing neuropathic pain.  This evidence suggests that gabapentin 
could be effective in the treatment of scarring alopecia, especially if increased CGRP is 
contributing to symptomatology. 
 
This study will serve as a pilot study to determine the efficacy and safety of topical 
gabapentin in the treatment of symptomatic scarring alopecia.  In this study, 10 subjects 
with symptomatic lymphocytic-type scarring alopecia will be recruited and treated with 
topical gabapentin.  Disease burden will be evaluated before and after 12 weeks of 
treatment through reporting of subjective symptomatology via surveys/questionnaire, 
neurometer study, clinical assessment, and biopsies measuring levels of CGRP before 
and after treatment.  
 
1.5 
Dose Rationale and Risk/Benefits 
The maximum daily dose of topical gabapentin will be 2 mL daily. The drug will be 
administered topically by the subject to symptomatic affected areas of intact skin two 
times daily for 12 weeks. Subjects will be seen in follow up on Week 8 and Week 12 
(Visits 4 and 5). 
 
Gabapentin will be compounded into a 6% solution based on a preparation method 
described by Boardman et al. (5) and approved by Investigational Drug Services (IDS) 
Pharmacy, University of Minnesota. IDS will prepare and dispense the drug.   
 
From published data in which gabapentin was used in a cohort group with nerve-related 
symptoms from localized or generalized vulvodynia, the most frequently reported 
adverse effect was irritation at the site of application, which led to termination of use 
(8%). Common adverse effects of oral gabapentin, including dizziness, somnolence, 
and peripheral edema, were not reported by any of the patients studied with vulvodynia. 
In this pilot study for scarring alopecia, the maximum daily dose will not exceed 2 mL of 
topical gabapentin 6% solution to affected scalp surface.    

 
 
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Based on its half-life of 8 hours, gabapentin will be administered two times a day during 
waking hours and the dose will not exceed the amount of a topical agent used on 
average to treat the entire scalp in everyday clinical practice (60 ml per month). 
 
Subjects may directly benefit from analysis of nerve function, CGRP neuropeptide, and 
treatment with gabapentin, resulting in improvement of their symtoms that may prevent 
further hair loss and even possible hair growth. This analysis will also help us better 
understand the disease process in scarring alopecia to further improve management of 
the condition.   
 
2 
Study Objectives 
Primary Objective: To assess the role of neurogenic inflammation associated with 
CGRP expression in scarring alopecia   
 
Secondary Objective: To assess the safety and efficacy of topical 6% gabapentin in 
subjects with symptomatic scarring alopecia.  
 
3 
Study Design 
 
3.1 
General Design 
This is a cohort study.  
 
Every subject is expected to schedule 6 visits total to complete the study. The total 
duration of the study for each volunteer is expected to be approximately 14 weeks. 
 
Prior to gabapentin treatment, there is one visit to screen individuals for participation in 
the study and to obtain consent. After consent is signed, this visit will also include 
obtaining history from the subject, clinical evaluation, survey completion, neurometer 
study, photography, and scalp biopsies. At this initial visit, a total of four 4-mm skin 
biopsies will be collected. Two biopsies will be collected from affected, symptomatic 
scalp, and two skin biopsies will be collected from clinically unaffected scalp. Routine 
histology, confocal microscopy examination, and, if indicated, ELISA CGRP level 
analysis will be performed on the biopsy specimens. Subjects will start topical 6% 
gabapentin treatment on Visit 2 (Day 0).  Four remaining visits will be scheduled to 
follow subjects after the start of topical 6% gabapentin treatment.  Visits 3, 4 and 5 will 
occur 4, 8 and 12 weeks respectively after treatment with gabapentin is started. These 
visits will include labwork to check gabapentin blood levels. During Visit 5, the subject 
will also be reassessed through clinical evaluation, completion of surveys, neurometer 
study, photography, and four 4-mm scalp biopsies. Visit 6 will be a follow-up visit two 
weeks after completion of treatment for suture removal and clinical evaluation of the 
subject. 

 
 
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TABLE 1. Schedule of Study Procedures 
Schedule of Study Procedures 
Visit 1 
Visit 2 
Visit 3 
Visit 4 
Visit 5 
Visit 6 
Screening/ 
Baseline 
 
 
 
 
Follow-up 
 
Day -14 
Day 0 
Week 4 
Week 8 
Week 12 
Week 14 
Approximate visit duration 
2-4 hours 
30 min 
30 min 
30 min 
1-2 hours 
20 min 
Assess inclusion/exclusion 
criteria, obtain informed consent 
X 
  
  
  
  
  
Obtain medical history 
 
 
 
 
 
 
Physical examination of scalp 
X 
  
  
 X 
  
Review changes in medical 
history and medications 
 
 
 
 
 
 
Surveysa 
X 
  
X 
 X 
 X 
  
Neurometer studyb 
X 
  
  
 X 
  
Record adverse experiences 
  
  
X 
X 
X 
X 
LPPAI clinical assessmentc 
X 
X 
X 
X 
X 
  
Gabapentin blood level 
 
 
X 
X 
X 
 
Skin biopsies 
X 
 
 
 
X 
 
Biopsy suture removal 
 
X 
 
 
 
X 
Photographsd 
X 
  
  
 X 
  
Topical gabapentin 6% solution 
BID to affected area 
  
X 
X 
X 
 
 
aSurveys include the Dermatology Life Quality Index (DLQI), Short Form (36) Health Survey (SF-36), Pain 
Visual Analog Scale (VAS) and Itch Visual Analog Scale (VAS). 
bNeurometer study will be performed on three areas: the index finger; normal-appearing, unaffected 
scalp;  affected symptomatic scalp. 
cLichen Planopilaris Activity Index (LPPAI) clinical assessment was developed by Chiang et al. and 
published in JAAD in January 201024. 
dPhotographs will focus on the affected symptomatic areas of the scalp at Visit 1.  Photographs of the 
same areas will be taken at Visit 5. 
 
3.2 
Primary Study Endpoints 
(1) To assess the role of neurogenic inflammation in symptomatic scarring alopecia and 
(2) assess the efficacy of topical 6% gabapentin in the management of symptomatic 
scarring alopecia patients. Subjects’ disease burden will be assessed by clinical exam, 
qualitatively with surveys and neurometer study, and skin biopsies to confirm disease 
diagnosis, assess disease inflammation, and measure CGRP levels. Subjects will begin 
topical 6% gabapentin treatment for 12 weeks starting at Visit 2. They will be followed 
up every 4 weeks during treatment and will be reassessed at week 12 (visit 5) with 
clinical exam, qualitatively with surveys and neurometer study, and final biopsies to 
evaluate the impact of gabapentin on the disease and CGRP levels. The surveys to be 
filled out by the subjects include the Dermatology Life Quality Index (DLQI), Short Form 
(36) Health Survey (SF-36), Visual Analog Scale for Pain, and Visual Analog Scale for 
Itch. 
 

 
 
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3.3 
Secondary Study Endpoints 
To assess the safety, tolerability, and efficacy of topical gabapentin for 12 weeks in 
subjects with lymphocytic-type symptomatic scarring alopecia. Subjects will be followed 
up 4, 8, and 12 weeks during treatment with topical 6% gabapentin starting day 0 (Visit 
2) and ending week 12 (Visit 5). Medication side effects, blood levels, and adverse 
experiences will be recorded. 
 
3.4 
Primary Safety Endpoints 
A primary safety endpoint will take place when the first 5 subjects complete the study. 
 
4 
Subject Selection and Withdrawal 
 
4.1 
Inclusion Criteria 
1. 
Male and female adults, greater than 18 years of age 
2. 
Biopsy-proven diagnosis of primary scarring alopecia of lymphocytic 
inflammatory infiltrate type, indicated as one of the following conditions: lichen 
planopilaris, frontal fibrosing alopecia, or central centrifugal cicatricial alopecia  
3. 
At least one persistent scalp symptom associated with inflammation: pain, 
burning, itch, tingling/crawling, stinging, or tenderness 
4. 
Able to complete survey and questionnaire subjectively 
5. 
Consents to participate in neurometer study and scalp biopsy acquisition 
6. 
Willingness to adhere to study protocol 
7. 
If subject is taking a neuromodulatory medication (including capsaicin cream, 
tricyclic antidepressants, carbamazepine, phenytoin, topiramate, 
oxcarbazepine, lamotrigine, morphine, Botox, etc), he or she must be a stable 
dose for at least 6 months prior to study enrollment 
 
The gender of the subjects will not determine enrollment.  There is no requirement of 
male to female ratio for this study. The age range of the subjects will be 18 years and 
older. No specific racial or ethnic restrictions will be present for this study. The FDA lists 
gabapentin as Pregnancy Category C: studies in animals have shown some harm to the 
fetus but its effects on human fetus are unknown due to limited data. It has not been 
found to be harmful during breastfeeding. Therefore, female subjects of childbearing 
potential will not be excluded since the drug will be topically administered with minimal 
systemic absorption.  
 
4.2 
Exclusion Criteria 
1. 
Allergy or intolerance to gabapentin or the substances used in its 
compounding  
2. 
Underlying disease that might be adversely affected by topical gabapentin  
3. 
Application of topical immunomodulatory or immunosuppressive agent to the 
scalp in the preceding 2 weeks 
4. 
Systemic administration of corticosteroid or other systemic treatment (i.e., 
methotrexate, phototherapy) that has immunomodulatory or other 
immunosuppressive mechanism of action, in the preceding 8 weeks 

 
 
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5. 
Clinical evidence of secondary skin infection 
6. 
Individuals who have undergone scalp reduction surgery or hair 
transplantation 
7. 
Asymptomatic disease  
8. 
Immunosuppression due to disease state or use of systemic/topical biological 
agents (HIV, chemotherapy, immunomodulators, history of transplantation) 
9. 
Any Investigational medications within the past 30 days, including those for 
migraines or scarring alopecias (anti-CGRP agents) 
10. Use of GABAergic medications (including gabapentin and pregabalin) in the 
preceding 2 months 
11. Use of illicit drugs or opioid medications 
12. Evidence of anemia, thyroid disease, sarcoidosis or other medical condition 
that could impact hair growth and adversely impact the outcome of the study 
13. Implantable Cardioverter Defibrillator (ICD) or pacemaker 
14. Subject has any medical condition that, in the judgment of the Investigator, 
would jeopardize the subject’s safety following exposure to the administered 
medications 
 
If subjects are taking medications/treatments for their scarring alopecia at baseline visit, 
they will be asked to stop taking/administering them as described in the exclusion 
criteria. 
 
 
4.3 
Subject Recruitment and Screening 
Subjects will be identified by the Principal Investigator, Dr. Maria K. Hordinsky. The 
study coordinator will assist in describing the study and answering questions. 
 
Advertisements may be posted on the University of Minnesota campus and surrounding 
community. Advertising will be submitted to the Institutional Review Board (IRB) for 
approval before it is used. Language will also be submitted to the IRB so information 
about the study can be posted on our department website, as well distributed to the 
Cicatricial Alopecia Research Foundation. 
 
In the screening process (Visit 1), subjects will be evaluated using the inclusion and 
exclusion criteria.  
   
 
4.4 
Early Withdrawal of Subjects 
 
4.4.1 
When and How to Withdraw Subjects 
Participation in this study is voluntary. Subjects are free to withdraw at any time. Once 
initiated, individual subjects will receive enough gabapentin for 4 weeks at a time to 
administer. If withdrawn, subject will be asked to terminate use of the investigational 
drug and return the remaining gabapentin solution.  
 

 
 
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Investigators may withdraw subjects from the study if subjects fail to adhere to the study 
protocol requirements (e.g. use of other topical, oral or other medical treatments used 
for management of scarring alopecia during the study). Subjects may also be withdrawn 
if they experience an adverse reaction to the study medication or if their gabapentin 
blood levels exceed safety levels (>16 ug/mL). 
 
4.4.2 
Data Collection and Follow-up for Withdrawn Subjects 
Data collection of withdrawn subjects will be collected up to the point of withdrawal. This 
data will still be included in the pool-data of the study for further analysis. Subjects who 
withdraw from further data collection will not be replaced with additional subjects.  
 
If a subject withdraws consent to participate in the study, attempts will be made to 
obtain permission to record data up to the protocol-described end of subject follow-up 
period. 
 
Three phone calls one week apart one from another will be made to the subjects lost to 
follow-up to record data up to the protocol-described end of subject follow-up period. 
 
5 
Study Drug  
 
5.1 
Description 
Gabapentin is a GABA analogue anticonvulsant also used to treat neuropathic pain. It is 
thought to target voltage-gated calcium ion channels at pre- and post-synaptic 
membranes of neurons in the central and peripheral nervous system modulating the 
release of neurotransmitters.  
 
Gabapentin is normally available in solid (powder) form and must be compounded into a 
topical solution. Based on published data of effective topical gabapentin preparation in 
vulvodynia and experience preparing topical gabapentin for use in patients with pain, 
IDS Pharmacy of University of Minnesota will compound the gabapentin into the 
solution for topical use. Once prepared, IDS will also be responsible for dispensing the 
solution of 6% gabapentin in an appropriate drug container to be given to subjects. 
Each subject participating in the study will receive enough gabapentin solution for use 
over four weeks, approximately a maximum of 2 mL a day; 60 mL for 4 weeks. After 4 
weeks of treatment (Visit 4) and 8 weeks of treatment (Visit 5) the subject will return for 
follow up with the prescribed gabapentin container and will be given a new dispensed 
bottle of the solution for use during the next four weeks.    
 
Treatment Regimen 
Qualified subjects will be educated by the research coordinator on how to properly apply 
the gabapentin 6% solution two times daily onto intact skin of affected symptomatic 
scalp areas.  The minimum and the maximum size of the scalp areas to be treated will 
be determined for each subject respectively, based on their disease pattern assessed 
during Visit 2.  Treated sites will be initially recorded and should be treated for the entire 
12 weeks unless the skin is adversely affected by the topical gabapentin.    
 

 
 
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Subjects may continue their normal grooming regimen as long as substances without 
other neuromodulatory agents or steroids are used during the treatment period. 
Recommended application will be in morning and evening at bedtime. The solution 
should be applied after hair washing if this coincides with time for application.   
 
5.2 
Method for Assigning Subjects to Treatment Groups 
All participants will receive treatment with topical 6% gabapentin. The study design does 
not include a control group, therefore, randomization of participants will not occur.   
 
5.3 
Preparation and Administration of Study Drug 
IDS will be responsible for the preparation and dispensing of gabapentin 6% solution to 
the principal investigators or clinical research coordinator (study team). The clinical 
research coordinator will provide subjects with the solution to administer two times daily 
after educating them on how to apply the drug. The gabapentin solution should be 
stored at room temperature (20° to 25°C)/(68° to 77°F) while always properly sealed 
and away from light and moisture. The study team will provide the subjects with a four 
week supply of solution at a time. Subjects will contact investigators or coordinator if 
they need more solution and will be asked to do so promptly if low on solution to avoid 
gaps in treatment.   
 
5.4 
Subject Compliance Monitoring 
Subject participants will be informed that they will be asked to stop taking any 
medications/treatments for their scarring alopecia while participating in this study.  This 
statement will be also written in the consent form that subjects will be asked to read, 
understand and sign prior participating in the study.  
  
5.5 
Prior and Concomitant Therapy 
Subjects will be asked detailed information regarding their medical and medication 
history for general health issues and specific focused questions regarding their 
symptomatic scarring alopecia.  
 
Subjects will not be allowed to use any medicines/therapies other than the gabapentin 
6% solution on the scalp for their symptomatic scarring alopecia. 
 
5.6 
Packaging 
The gabapentin 6% solution will be prepared through a compounding method by IDS 
Pharmacy and will be dispensed in red sealed containers with 60 mL of solution.   
 
5.7 
Blinding of Study Drug 
This is not a blinded study.  
 
5.8 
Receiving, Storage, Dispensing and Return 
 

 
 
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5.8.1 
Receipt of Drug Supplies 
The Department of Dermatology Clinical Research Division will order 10 initial 
containers of 60 mL solution of gabapentin 6% solution from the IDS Pharmacy, 
University of Minnesota, Mayo Building, 2nd floor.  
 
Upon receipt of the study treatment supplies, an inventory will be performed and a drug 
receipt log filled out and signed by the person accepting the shipment.  It is important 
that the designated study staff counts and verifies that the shipment contains all the 
items noted in the shipment inventory.  Any damaged or unusable study drug in a given 
shipment will be documented in the study files.   
 
Prepared containers of gabapentin solution 6% will be kept at room temperature (20° to 
25°C) away from moisture and light for up to 24 months at the IDS pharmacy, University 
of Minnesota, Phillips Wangensteen Building, 2nd floor.  
 
5.8.2 
Dispensing of Study Drug 
IDS will dispense the drug-filled container to the investigators or coordinators. Once 
qualified to begin treatment, each subject will receive one container of 60 mL of 
gabapentin 6% solution from the clinical coordinator.  Regular study drug reconciliation 
will be performed to document drug assigned. During follow-up visits, drug consumed 
and drug remaining will be recorded. This reconciliation will be logged on the drug 
reconciliation form, and signed and dated by the study team. 
 
5.8.3 
Return or Destruction of Study Drug 
At the completion of the study, there will be a final reconciliation of drug dispensed, drug 
consumed, and drug remaining. This reconciliation will be logged on the drug 
reconciliation form, signed and dated. Any discrepancies noted will be investigated, 
resolved, and documented prior to return or destruction of unused study drug. Drug 
destroyed on site will be documented in the study files. 
 
6 
Study Procedures 
 
6.1 
Visit 1 (Screening/baseline) 
Before any study-related procedures are performed, subjects will be screened for 
participation in the study using inclusion and exclusion criteria.  Once determined to be 
a qualified candidate for participation, subjects will be asked to read and sign the 
consent form if they wish to participate in this research study. They will then be asked 
about their medical and medication history.  
 
The following tests and procedures will be performed: 
 Obtain medical history and medications 
 Physical examination of scalp 
 Lichen Planopilaris Activity Index (LPPAI) clinical assessment will be performed. 
Every effort will be made to have the same person evaluate the alopecia at each 
visit for any given subject.   

 
 
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 Scalp photography, especially affected areas that will be monitored and treated 
with gabapentin 
 Subject to complete surveys to assess symptomatology pre-treatment: DLQI, SF-
36, Pain VAS and Itch VAS 
 Neurometer study to assess Aβ, Aδ, and C fiber function before gabapentin 
administration 
 Collect four pre-treatment skin biopsy samples: two 4-mm biopsy specimens 
from affected scalp and two 4-mm biopsy specimens from normal-appearing 
scalp. Specimens will be analyzed for routine histology, confocal microscopic 
examination, and CGRP level.  
 
If potential symptomatic subjects are currently taking medications/treatments for their 
scarring alopecia, they will be asked to stop the treatment as noted in the exclusion 
criteria in section 4.2. 
6.2 
Visit 2 (Day 0) 
The following tests and procedures will be performed: 
 Record adverse experiences 
 Biopsy suture removal 
 Review changes in medical history and medications since Visit 1 
 LPPAI clinical assessment 
 Scalp photography, especially affected areas that will be monitored and treated 
with gabapentin 
 Subject to complete surveys to assess symptomatology pre-treatment: DLQI, SF-
36, Pain VAS and Itch VAS  
 Educate subject on how to apply topical gabapentin solution 
 Provide subject with first container of gabapentin solution 
 Subject begins treatment with topical gabapentin solution 
6.3 
Visit 3 (Week 4)  
The following tests and procedures will be performed: 
 Record adverse experiences 
 Gabapentin blood level 
 Review changes in medical history and medications since Visit 2 
 LPPAI clinical assessment 
 Subject to complete surveys to assess symptomatology: DLQI, SF-36, Pain VAS 
and Itch VAS 
 Collect and assess first container of gabapentin solution 
 Provide subject with new container of gabapentin solution 
 
Subject has now completed four weeks of topical gabapentin therapy. Answer any 
questions/concerns subject has about drug application and collect feedback on ease of 
use and/or impact on grooming practices. 

 
 
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6.4 
Visit 4 (Week 8)  
The following tests and procedures will be performed: 
 Record adverse experiences 
 Gabapentin blood level 
 Review changes in medical history and medications since Visit 3 
 LPPAI clinical assessment 
 Subject to complete surveys to assess symptomatology: DLQI, SF-36, Pain VAS 
and Itch VAS 
 Collect and assess second container of gabapentin solution 
 Provide subject with new container of gabapentin solution 
 
Subject has now completed eight weeks of topical gabapentin therapy. Answer any 
questions/concerns subject has about drug application and collect feedback on ease of 
use and/or impacting on grooming practices. 
6.5 
Visit 5 (Week 12) 
The following tests and procedures will be performed: 
 Record adverse experiences 
 Physical examination of scalp 
 Gabapentin blood level 
 Review changes in medical history and medications since Visit 4 
 LPPAI clinical assessment 
 Scalp photography, especially affected areas that are being monitored and 
treated with gabapentin 
 Subject to complete surveys to assess symptomatology: DLQI, SF-36, Pain VAS 
and Itch VAS 
 Neurometer study to assess Aβ, Aδ, and C fiber function after gabapentin 
administration 
 Collect and assess third container of gabapentin solution 
 Collect four post-treatment skin biopsy samples: two 4-mm skin biopsy 
specimens from affected scalp and two 4-mm biopsy specimens from normal-
appearing scalp. Specimens will be analyzed for routine histology, confocal 
microscopic examination, and CGRP level.  
 
Subject has now completed the entire twelve weeks of topical gabapentin therapy. 
6.6 
Visit 6 (2 week follow-up) 
The following tests and procedures will be performed: 
 Record adverse experiences 
 Review changes in medical history and medications since Visit 5 
 LPPAI clinical assessment 
 Subject to complete surveys to assess symptomatology: DLQI, SF-36, Pain VAS 
and Itch VAS 
 Biopsy suture removal 

 
 
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A flowchart of the study can be found in Section 3.1 General Design. 
 
7 
Statistical Plan 
 
7.1 
Sample Size Determination 
This is a pilot study with the plan to enroll 10 subjects. The reasoning for sample size 
selection is to proceed with preliminary studies in a small group. All subjects will be 
selected based on inclusion and exclusion criteria. 
 
7.2 
Statistical Methods 
An intent-to-treat analysis will be performed in all subjects for symptoms and sign 
scores comparing the last treatment visit to baseline. A combination of methods will be 
included for analysis. Descriptive statistics including either numerically or graphical 
representations to summarize the data will be used. 
 
7.3 
Subject Population(s) for Analysis 
Data collected from all subjects will be included for the analysis. Data collection of 
withdrawn subjects will be collected up to the point of withdrawal. This data will still be 
included in the pool-data of the study for further analysis. 
 
8 
Safety and Adverse Events 
 
8.1 
Definitions 
Adverse Event 
An adverse event (AE) is any symptom, sign, illness or experience that develops or 
worsens in severity during the course of the study. Intercurrent illnesses or injuries 
should be regarded as adverse events.  Abnormal results of diagnostic procedures are 
considered to be adverse events if the abnormality: 
 results in study withdrawal 
 is associated with a serious adverse event 
 is associated with clinical signs or symptoms 
 leads to additional treatment or to further diagnostic tests 
 is considered by the investigator to be of clinical significance 
 
Serious Adverse Event 
Adverse events are classified as serious or non-serious.  A serious adverse event is 
any AE that is:  
 fatal 
 life-threatening 
 requires or prolongs hospital stay 
 results in persistent or significant disability or incapacity 
 a congenital anomaly or birth defect 
 an important medical event 

 
 
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Important medical events are those that may not be immediately life threatening, but are 
clearly of major clinical significance.  They may jeopardize the subject, and may require 
intervention to prevent one of the other serious outcomes noted above. For example, 
drug overdose or abuse, a seizure that did not result in in-patient hospitalization, or 
intensive treatment of bronchospasm in an emergency department would typically be 
considered serious.  
 
All adverse events that do not meet any of the criteria for serious will be regarded as 
non-serious adverse events.  
 
Adverse Event Reporting Period 
The study period during which adverse events must be reported is normally defined as 
the period from the initiation of any study procedures to the end of the study treatment 
follow-up.  For this study, the study treatment follow-up is defined as 14 weeks following 
the start of administration of study treatment (Day 0).  
 
Preexisting Condition 
A preexisting condition is one that is present at the start of the study.  A preexisting 
condition will be recorded as an adverse event if the frequency, intensity, or the 
character of the condition worsens during the study period. 
 
General Physical Examination Findings 
At screening, any clinically significant abnormality will be recorded as a preexisting 
condition. At the end of the study, any new clinically significant findings/abnormalities 
that meet the definition of an adverse event will also be recorded and documented as 
an adverse event.  
 
Post-study Adverse Event 
All unresolved adverse events will be followed by the investigator until the events are 
resolved, the subject is lost to follow-up, or the adverse event is otherwise explained.  At 
the last scheduled visit, the investigator will instruct each subject to report any 
subsequent event(s) that the subject, or the subject’s personal physician, believes might 
reasonably be related to participation in this study.   
 
Abnormal Laboratory Values 
Gabapentin blood levels will be monitored to ensure gabapentin toxicity does not occur.  
If gabapentin levels reach or exceed 16 ug/mL, gabapentin treatment will be interrupted.  
Abnormal gabapentin levels will be recorded as an adverse event and followed by the 
investigator until the event has resolved. 
 
Hospitalization, Prolonged Hospitalization or Surgery 
Any adverse event that results in hospitalization or prolonged hospitalization will be 
documented and reported as a serious adverse event unless specifically instructed 
otherwise in this protocol.  Any condition responsible for surgery will be documented as 
an adverse event if the condition meets the criteria for an adverse event.  

 
 
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Neither the condition, hospitalization, prolonged hospitalization, nor surgery are 
reported as an adverse event in the following circumstances: 
 Hospitalization or prolonged hospitalization for diagnostic or elective surgical 
procedures for a preexisting condition.  Surgery should not be reported as an 
outcome of an adverse event if the purpose of the surgery was elective or 
diagnostic and the outcome was uneventful. 
 Hospitalization or prolonged hospitalization required to allow efficacy 
measurement for the study.  
 Hospitalization or prolonged hospitalization for therapy of the target disease of 
the study, unless it is a worsening or increase in frequency of hospital 
admissions as judged by the clinical investigator. 
 
8.2 
Recording of Adverse Events 
At each contact with the subject, the investigators will seek information on adverse 
events by specific questioning and, as appropriate, by examination.  Information on all 
adverse events will be recorded immediately in the source document, and also in the 
appropriate adverse event module of the case report form (CRF).  All clearly related 
signs, symptoms, and abnormal diagnostic procedures results will be recorded in the 
source document, though will be grouped under one diagnosis. 
 
All adverse events occurring during the study period will be recorded.  The clinical 
course of each event will be followed until resolution, stabilization, or until it has been 
determined that the study treatment or participation is not the cause.  Serious adverse 
events that are still ongoing at the end of the study period will be followed up to 
determine the final outcome.  Any serious adverse event that occurs after the study 
period and is considered to be possibly related to the study treatment or study 
participation will be recorded and reported immediately. 
 
8.3 
Reporting of Serious Adverse Events 
 
8.3.1 
Study Sponsor Notification by Investigator 
A Serious Adverse Event (SAE) form will be completed by the investigator within 24 
hours. The investigator will keep a copy of this SAE form on file at the study site.  
Report serious adverse events by phone and facsimile to: 
 
 
Maria K. Hordinsky, MD     
 
Cell: 612-710-5507     
 
Fax: 612-624-6678 
 
At the time of the initial report, the following information should be provided: 

 
 
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 Study identifier 
 Study Center 
 Subject number 
 A description of the event 
 Date of onset 
 Current status 
 Whether study treatment was 
discontinued 
 The reason why the event is 
classified as serious 
 Investigator assessment of the 
association between the event and 
study treatment 
 
Within the following 48 hours, the investigator will provide further information on the 
serious adverse event in the form of a written narrative. This should include a copy of 
the completed Serious Adverse Event form, and any other diagnostic information that 
will assist the understanding of the event. Significant new information on ongoing 
serious adverse events will be provided promptly to the principal investigator, Maria K. 
Hordinsky, and Department of Dermatology. 
 
8.3.2 
IRB Notification by Investigator 
Reports of all serious adverse events (including follow-up information) will be submitted 
to the IRB within 10 working days if it falls under the UPIRTSO guidelines.  Copies of 
each report and documentation of IRB notification and receipt will be kept in the Clinical 
Investigator’s binder. 
 
8.3.3 
FDA Notification by Sponsor 
The study sponsor shall notify the FDA by telephone or by facsimile transmission of any 
unexpected fatal or life-threatening experience associated with the use of the drug as 
soon as possible but no later than 7 calendar days from the investigator’s original 
receipt of the information. 
 
If a previous adverse event that was not initially deemed reportable is later found to fit 
the criteria for reporting, the investigators will submit the adverse event in a written 
report to the FDA as soon as possible, but no later than 15 calendar days from the time 
the determination is made. 
 
8.4 
Unblinding Procedures 
This is not a blinded study. 
 
8.5 
Stopping Rules  
Individual subjects will be administering topical gabapentin independently without 
supervision by study team.  Subjects are to terminate treatment if any new adverse 
symptoms different from before treatment occur or worsen and contact the study team 
immediately.  Individual subjects will also be asked to terminate treatment if their 
gabapentin blood levels are >16 ug/mL. 
 
8.6 
Medical Monitoring 
It is the responsibility of the Principal Investigator to oversee the safety of the study at 
his/her site.  This safety monitoring will include careful assessment and appropriate 
reporting of adverse events as noted above, as well as the construction and 

 
 
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implementation of a site data and safety-monitoring plan (see section 10 Auditing, 
Monitoring and Inspecting).  Medical monitoring will include a regular assessment of the 
number and type of serious adverse events. 
 
9 
Data Handling and Record Keeping 
 
9.1 
Confidentiality 
Information about study subjects will be kept confidential and managed according to the 
requirements of the Health Insurance Portability and Accountability Act of 1996 
(HIPAA).  Those regulations require a signed subject authorization informing the subject 
of the following:  
 
 What protected health information (PHI) will be collected from subjects in this 
study 
 Who will have access to that information and why 
 Who will use or disclose that information 
 The rights of a research subject to revoke their authorization for use of their PHI  
 
In the event that a subject revokes authorization to collect or use PHI, the investigator, 
by regulation, retains the ability to use all information collected prior to the revocation of 
subject authorization.  For subjects that have revoked authorization to collect or use 
PHI, attempts should be made to obtain permission to collect at least vital status (i.e. 
that the subject is alive) at the end of their scheduled study period. 
 
9.2 
Source Documents/Case Report Forms 
Source data for this study include subject’s medical records, , surveys (DLQI, SF-36, 
Pain VAS, Itch VAS), photographs, neurometer study data, biopsy pathology reports, 
gabapentin level lab reports, ELISA CGRP level reports, pharmacy dispensing records, 
confocal microscopy photographs, and subject files. 
 
Data generated by the methods described in the protocol will be recorded in the 
subjects' medical records and/or study progress notes. Data may be transcribed legibly 
on CRFs supplied for each subject or directly inputted into an electronic system or any 
combination thereof. 
 
9.3 
Records Retention 
Investigators will retain study essential documents for at least 2 years after the formal 
discontinuation of the study.  The documents will be locked and stored in the clinical 
research office space.  
 
10 
Study Auditing, and Inspecting 
 
10.1 
Auditing and Inspecting 
The investigator will permit study-related monitoring, audits, and inspections by the IRB, 
the sponsor, government regulatory bodies, and University compliance and quality 

 
 
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assurance groups of all study related documents (e.g. source documents, regulatory 
documents, data collection instruments, study data etc.).  The investigator will ensure 
the capability for inspections of applicable study-related facilities (e.g. pharmacy, 
diagnostic laboratory, etc.). 
 
Participation as an investigator in this study implies acceptance of potential inspection 
by government regulatory authorities and applicable University compliance and quality 
assurance offices. 
 
11 
Ethical Considerations 
This study is to be conducted according to US and international standards of Good 
Clinical Practice (FDA Title 21 part 312 and International Conference on Harmonization 
guidelines), applicable government regulations and Institutional research policies and 
procedures. 
 
This protocol and any amendments will be submitted to a properly constituted 
independent Institutional Review Board (IRB), in agreement with local legal 
prescriptions, for formal approval of the study conduct.  The decision of the IRB 
concerning the conduct of the study will be made in writing to the investigator and a 
copy of this decision will be provided to the sponsor before commencement of this 
study.   
 
All subjects for this study will be provided a consent form describing this study and 
providing sufficient information for subjects to make an informed decision about their 
participation in this study.  The consent form will be submitted with the protocol for 
review and approval by the IRB for the study.  The formal consent of a subject, using 
the IRB-approved consent form, will be obtained before that subject is submitted to any 
study procedure.  This consent form will be signed by the subject or legally acceptable 
surrogate, and the investigator-designated research professional obtaining the consent.  
 
12 
Study Finances 
 
12.1 
Funding Source 
The Cicatricial Alopecia Research Foundation will fund the treatment portion of the 
study. The Department of Dermatology Clinical Research Division, University of 
Minnesota, will fund the biopsy portion of the study with internal funding. Subjects 
and/or their insurance companies will not be charged for study-related procedures or 
visits. 
 
12.2 
 Research Related Injury 
In the event that this research activity results in an injury, treatment will be available,  
including first aid, emergency treatment and follow-up care as needed. Care for such 
injuries will be billed in the ordinary manner, to the subject or the subject's insurance 
company.  

 
 
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12.3 
Subject Stipends or Payments 
Subjects will receive $25.00 for each biopsy obtained as part of the study. Payment will 
be provided in the form of a gift card after acquisition of the scalp biopsies at Visits 1 
and 5.   
 
12.4 
Conflict of Interest 
The investigators have no conflict of interest. 
 
Any investigator who has a conflict of interest with this study (patent ownership, 
royalties, or financial gain greater than the minimum allowable by their institution, etc.) 
will refer to the Regents Policies on Individual Conflict of Interest Policy or Institutional 
Conflict of Interest Policy. These policies require University Faculty and staff to report 
external professional activities and business and significant financial interests related to 
his or her University activities by submitting a REPA (Report of External Professional 
Activities) at least once per year. Faculty and staff will also file a REPA when substantial 
changes in business or financial interests occur, when an activity that presents a 
potential conflict of interest is anticipated, or when submitting an application for research 
support or technology transfer, submitting research protocols to the IRB, or receiving 
financial contributions. All University of Minnesota investigators will follow the University 
conflict of interest policy. 
 
13 
Publication Plan 
Maria Hordinsky, MD holds the primary responsibility for publication of any results of the 
study.  Other investigators must first obtain approval from the primary responsible party 
before any information may be used or passed on to a third party. 
 
14 
References 
1. Harries MJ, Sinclair RD, MacDonald-Hull S, Whiting DA, Griffiths CEM, Paus R. 
Management of primary cicatricial alopecias: options for treatment. Br J Dermatol, 2008 
Jul; 159 (1):1-22. 
2. Hainline B.  Chronic pain: physiological, diagnostic, and management considerations. 
Psychiatr Clin N Am, 2005; 28: 713–735.  
3. Wacnik PW, Baker CM, Herron MJ, Kren BT, Blazar BR, Wilcox GL, Hordinsky MK, 
Beitz AJ, Ericson ME. Tumor-induced mechanical hyperalgesia involves CGRP 
receptors and altered innervation and vascularization of DsRed2 fluorescent hindpaw 
tumors. Pain, 2005 May;115(1-2):95-106.4).  
4. Durham PL. Inhibition of calcitonin gene-related peptide function: a promising 
strategy for treating migraine. Headache, 2008 Sep; 48(8):1269-75. 
5. Boardman LA, Cooper AS, Blais LR, Raker CA. Topical gabapentin in the treatment 
of localized and generalized vulvodynia. Obstetrics and Gynecology 2008 Sep; 
112(3):579-585. 

 
 
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This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
6. Vuković V, Lovrencić-Huzjan A, Bosnar-Puretić M Demarin V. The efficacy of 
gabapentin in migraine prophylaxis: an observational open label study. Acta Clin Croat 
2009 Jun;48(2):145-51. 
7. Hamada J.  Use of antiepileptic drugs for the preventive treatment of migraine. Brain 
Nerve. 2009 Oct;61(10):1117-24 
8. Chiang C, Sah D, Cho B, Ochoad BE, Price VH.  Hydroxychloroquine and lichen 
planopilaris: Efficacy and introduction of Lichen Planopilaris Activity Index scoring 
system.  J Amer Acad Dermatol 2010; 62(3): 387-392. 
9. Olsen EA, Bergfeld WF, Cotsarelis G, Price VH, Shapiro J, Sinclair R, Solomon A, 
Sperling L, Stenn K, Whiting DA.  Summary of North American Hair Research Society 
(NAHRS) - sponsored workshop on cicatricial alopecia, Duke University Medical Center, 
F. J Amer Acad Dermatol 2003; 48:103-10. 
 
15 
Attachments 
Appendix A: Dermatology Life Quality Index (DLQI) 
Appendix B: Short Form (36) Health Survey (SF-36) 
Appendix C: Visual Analog Scale (VAS) for pain 
Appendix D: Visual Analog Scale (VAS) for itch 
Appendix E: Lichen Planopilaris Activity Index (LPPAI) 
Appendix F: Adverse Event Report Form  
 
 
 
 
 
 
 
 
 
 
 
 

 
 
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Appendix A: Dermatology Quality of Life Index (DLQI) 
 
DERMATOLOGY LIFE QUALITY INDEX 
 
The aim of this questionnaire is to measure how much your skin problem has affected your life OVER 
THE LAST WEEK.  Please tickone box for each question. 
 
1. 
Over the last week, how itchy, sore,  
 
 
 
Very much 
3 
 
painful or stinging has your skin    
 
 
 
A lot 
 
2 
 
been?   
 
 
 
 
 
 
A little  
1 
 
 
 
 
 
 
 
 
 
Not at all 
0 
 
2. 
Over the last week, how embarrassed 
 
 
 
Very much 
3 
 
or self-conscious have you been because   
 
 
A lot 
 
2 
 
of your skin? 
 
  
 
 
 
 
A little  
1 
 
 
 
 
 
 
 
 
 
Not at all 
0 
 
3. 
Over the last week, how much has your  
 
 
 
Very much 
3 
 
skin interfered with you going 
 
  
 
 
A lot 
 
2 
 
shopping or looking after your home or   
 
 
A little  
1 
 
garden?        
 
 
 
 
 
 
Not at all 
0 
 
 
 
 
 
 
 
 
 
Not relevant  
0 
 
4. 
Over the last week, how much has your  
 
 
 
Very much 
3 
 
skin influenced the clothes  
 
 
 
 
A lot 
 
2 
 
you wear? 
 
 
 
 
 
 
A little  
1 
 
 
 
 
 
 
 
 
 
Not at all 
0 
 
 
 
 
 
 
 
 
 
Not relevant  
0 
 
5. 
Over the last week, how much has your  
 
 
 
Very much 
3 
 
skin affected any social or  
 
 
 
 
A lot 
 
2 
 
leisure activities? 
 
 
 
 
 
A little  
1 
 
 
 
 
 
 
 
 
 
Not at all 
0 
 
 
 
 
 
 
 
 
 
Not relevant  
0 
 
6. 
Over the last week, how much has your  
 
 
 
Very much 
3 
 
skin made it difficult for   
 
 
 
 
A lot 
 
2 
 
you to do any sport? 
 
 
 
 
 
A little  
1 
 
 
 
 
 
 
 
 
 
Not at all 
0 
 
 
 
 
 
 
 
 
 
Not relevant 
0 
 
7. 
Over the last week, has your skin prevented 
 
 
Yes 
 
3 
 
you from working or studying?  
 
 
 
No 
 
 
 
 
 
 
 
 
 
 
 
Not relevant  
0 
 
 
 
If "No", over the last week how much has   
 
 
A lot 
 
2 
 
your skin been a problem at  
 
 
 
 
A little  
1 
 
work or studying? 
 
 
 
 
 
Not at all 
0 
 
 
Page 1 score ______ 

 
 
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8. 
Over the last week, how much has your  
 
 
 
Very much 
3 
 
skin created problems with your   
 
 
 
A lot 
 
2 
 
partner or any of your close friends 
 
 
 
A little  
1 
 
or relatives?  
 
 
 
 
 
 
Not at all 
0 
 
 
 
 
 
 
 
 
 
Not relevant  
0 
 
9. 
Over the last week, how much has your  
 
 
 
Very much 
3 
 
skin caused any sexual   
 
 
 
 
A lot 
 
2 
 
difficulties? 
 
 
 
 
 
 
A little  
1 
 
 
 
 
 
 
 
 
 
Not at all 
0 
 
 
 
 
 
 
 
 
 
Not relevant  
0 
 
10. 
Over the last week, how much of a  
 
 
 
Very much 
3 
 
problem has the treatment for your 
 
 
 
A lot 
 
2 
 
skin been, for example by making  
 
 
 
A little  
1 
 
your home messy, or by taking up time?   
 
 
Not at all 
0 
 
 
 
 
 
 
 
 
 
Not relevant 
0
 
Please check you have answered EVERY question. Thank you.  
AY Finlay, GK Khan, April 1992 www.dermatology.org.uk, this must not be copied without the permission of the authors. 
 
 
 
  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Appendix B: Short Form (36) Health Survey (SF-36) 
 
Page 2 score ______ 
Page 1 score ______ + Page 2 score ______ = Total DLQI score ______ 

 
 
Page 23 
CONFIDENTIAL 
This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
RAND 36-Item Health Survey 1.0 Questionnaire Items 
1. In general, would you say your health is: 
1 Excellent 
2 Very Good 
3 Good 
4 Fair 
5 Poor 
 
2. Compared to one year ago, how would you rate your health in general now? 
 
1 Much better now than one year ago 
 
2 Somewhat better now than one year ago 
 
3 About the same 
 
4 Somewhat worse now than one year ago 
 
5 Much worse now than one year ago 
 
The following items are about activities you might do during a typical day.  Does your health 
now limit you in these activities?  If so, how much? 
(Check one number on each line) 
 
Yes, Limited 
a Lot 
 
Yes, Limited 
a Little 
 
No, Not 
limited at All 
 
3. Vigorous activities, such as running, 
lifting heavy objects, participating in 
strenuous sports 
1 
2 
3 
4. Moderate activities, such as moving a 
table, pushing a vacuum cleaner, 
bowling, or playing golf 
1 
2 
3 
5. Lifting or carrying groceries 
1 
2 
3 
6. Climbing several flights of stairs 
1 
2 
3 
7. Climbing one flight of stairs 
1 
2 
3 
8. Bending, kneeling, or stooping 
1 
2 
3 
9. Walking more than a mile 
1 
2 
3 
10. Walking several blocks 
1 
2 
3 
11. Walking one block 
1 
2 
3 
12. Bathing or dressing yourself 
1 
2 
3 

 
 
Page 24 
CONFIDENTIAL 
This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
During the past 4 weeks, have you had any of the following problems with your work or other 
regular daily activities as a result of your physical health? 
(Check One Number on Each Line) 
 
Yes 
 
No 
 
13. Cut down the amount of time you spent on work or 
other activities 
1 
2 
14. Accomplished less than you would like 
1 
2 
15. Were limited in the kind of work or other activities 
1 
2 
16. Had difficulty performing the work or other 
activities (for example, it took extra effort) 
1 
2 
During the past 4 weeks, have you had any of the following problems with your work or other 
regular daily activities as a result of any emotional problems (such as feeling depressed or 
anxious)? 
(Check One Number on Each Line) 
 
Yes 
 
No 
 
17. Cut down the amount of time you spent on work 
or other activities 
1 
2 
18. Accomplished less than you would like 
1 
2 
19. Didn't do work or other activities as carefully as 
usual 
1 
2 
 
20. During the past 4 weeks, to what extent has your physical health or emotional problems 
interfered with your normal social activities with family, friends, neighbors, or groups? 
(Check One Number) 
1 Not at all 
2 Slightly 
3 Moderately 
4 Quite a bit 
5 Extremely 
 

 
 
Page 25 
CONFIDENTIAL 
This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
 
21. How much bodily pain have you had during the past 4 weeks? 
(Check One Number) 
1 None 
2 Very mild 
3 Mild 
4 Moderate 
5 Severe 
5 Very severe 
 
22. During the past 4 weeks, how much did pain interfere with your normal work (including 
both work outside the home and housework)? 
(Check One Number) 
1 Not at all 
2 A little bit 
3 Moderately 
4 Quite a bit 
5 Extremely 
These questions are about how you feel and how things have been with you during the past 4 
weeks. For each question, please give the one answer that comes closest to the way you have 
been feeling. 
How much of the time during the past 4 weeks . . . 
(Check One Number on Each Line) 
 
All of 
the Time 
 
Most of 
the Time 
 
A Good 
Bit of 
the Time 
 
Some of 
the Time 
 
A Little 
of the 
Time 
 
None of 
the Time 
 
23. Did you feel full of pep? 
1 
2 
3 
4 
5 
6 
24. Have you been a very 
nervous person? 
1 
2 
3 
4 
5 
6 
25. Have you felt so down 
in the dumps that 
nothing could cheer you 
up? 
1 
2 
3 
4 
5 
6 
26. Have you felt calm and 
peaceful? 
1 
2 
3 
4 
5 
6 

 
 
Page 26 
CONFIDENTIAL 
This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
 
27. Did you have a lot of 
energy? 
1 
2 
3 
4 
5 
6 
28. Have you felt 
downhearted and blue? 
1 
2 
3 
4 
5 
6 
29. Did you feel worn out? 
1 
2 
3 
4 
5 
6 
30. Have you been a happy 
person? 
1 
2 
3 
4 
5 
6 
31. Did you feel tired? 
1 
2 
3 
4 
5 
6 
 
32. During the past 4 weeks, how much of the time has your physical health or emotional 
problems interfered with your social activities (like visiting with friends, relatives, etc.)? 
 
(Check One Number) 
1 All of the time 
2 Most of the time 
3 Some of the time 
4 A little of the time 
5 None of the time 
How TRUE or FALSE is each of the following statements for you. 
(Circle One Number on Each Line) 
 
Definitely 
True 
 
Mostly 
True 
 
Don't 
Know 
 
Mostly 
False 
 
Definitely 
False 
 
1. I seem to get sick a little 
easier than other people 
1 
2 
3 
4 
5 
2. I am as healthy as anybody I 
know 
1 
2 
3 
4 
5 
3. I expect my health to get 
worse 
1 
2 
3 
4 
5 
4. My health is excellent 
1 
2 
3 
4 
5 
RAND-36 score: ______ 

 
 
Page 27 
CONFIDENTIAL 
This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
Appendix C: Itch Visual Analog Scale (VAS) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

 
 
Page 28 
CONFIDENTIAL 
This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
Appendix D: Pain Visual Analog Scale (VAS) 
 
Draw a line anywhere on the scale that best represents the severity of your 
pain: 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

 
 
Page 29 
CONFIDENTIAL 
This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
Appendix E: Lichen Planopilaris Activity Index (LPPAI) 
 
 
A. Pruritis 
0   1   2 3   
B. Pain 
0   1   2 3   
C. Burning 
0   1   2 3   
D. Erythema 
0   1   2 3   
E. Perifollicular erythema 
0   1   2 3   
F. Perifollicular scale 
0   1   2 3   
Pull test 
Positive    Negative   
Spreading 
Yes     
No     
Crusting 
Yes     
No     
Pustules 
Yes     
No     
Dimensions/extent 
Yes     
No     
Loss follicular markings 
Yes     
No     
Tufting 
Yes     
No     
Telangiectasia 
Yes     
No     
Atrophy 
Yes     
No     
Pigment change 
Yes     
No     
Other skin, nail, mucous membrane 
Yes     
No     
 
 
 
 
 
 
LPPAI score = (A+B+C+D+E+F)/3 + 2.5(pull test) + 1.5(spread/2) 
Scale: 
0 = negative 
1 = + / - 
2 = + 
3 = ++,+++ 
 
Positive/Yes = 1 
Negative/No = 0 

 
 
Page 30 
CONFIDENTIAL 
This material is the property of the University of Minnesota.  Do not disclose or use except as authorized in writing by the study sponsor 
Appendix F: Adverse Event Report Form 
 
Subject ID: 
 
Subject initials: 
 
This form is a tool for study personnel to track adverse events.  Adverse events need to be 
summarized at the time of annual IRB renewal.  All sections may be filled out by the principal 
investigator or co-investigators.   
 
A.  Adverse Event Description:  
 
 Medication 
 Blood Draw 
 Other _________________________ 
 
B.  Date & Time of Adverse Event: 
 
 
Start Date:   
 /  
      /  
 
Start Time:               :  
 
 
 
End Date:  
 
 /  
      /  
 
End Time:                :  
 
C.  Treatment Needed? 
 
 
 
 Yes  
 No 
 
 
If yes, describe: __________________________________________________ 
 
D.  Outcome of the Experience:   
 
 Resolved  
 Continuing 
 Death 
 
E.  Severity of Experience:  
Grade  
 1 
 2 
 3 
 4   5 
 
 
Severity Scale 
 CTCAE 
 Other _________ 
 
F.  Was this a serious event? 
 
 
 Yes  
 No 
 
G. Was the event unanticipated? 
 
 Yes  
 No 
 
H. Was the event related to study procedures? 
 
 
 Definitely  Probably  Possibly 
 Unlikely 
 Unrelated 
 
 
 
 
 
 
 
If yes to F-H, report to IRB as a UPIRTSO. 
 
G. Was this event recorded on AE log? 
 Yes  
 No 
 
Additional Comments: