A PROspective, Observational, US-based Study Assessing Outcomes, Adverse Events, Treatment Patterns, and Quality of Life in Patients Diagnosed With Mycosis Fungoides Cutaneous T-cell Lymphoma and Treated With Valchlor®
Conditions: Mycosis Fungoides
Sponsor: Helsinn Therapeutics (U.S.), Inc

 
 
 
TPL-100272 v01 
 
 
Mycosis fungoides cutaneous T-cell lymphoma 
Protocol AC-079A501 
PROVe 
A PROspective, observational, US-based study assessing outcomes, adverse events, 
treatment patterns, and quality of life in patients diagnosed with mycosis fungoides 
cutaneous T-cell lymphoma and treated with Valchlor® 
Authors: 
Badri Rengarajan, Carol Zhao, Sandy Petersen-Stenger 
Document type: 
Protocol for Non-Drug Interventional and Non-interventional 
(Observational) Studies using Epidemiological Methods 
Document status and 
version: 
Final Version 3 
Date: 
13 December 2016 
Number of pages: 
43 
Document number: 
D-16.626 
 
Confidentiality Statement 
The information contained in this document, especially unpublished data, is the property 
of Actelion Pharmaceuticals US, Inc. It is provided to third parties in confidence, and it is 
understood that this information will not be disclosed to any third party without written 
authorization from the data owner, as laid out in the respective agreement between the 
parties.  

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CONTACT DETAILS 
 
Sponsor (Data Owner) 
Actelion Pharmaceuticals US, Inc. 
5000 Shoreline Court, Suite 200 
South San Francisco, CA 94080 
Clinical Study Leader/Project 
Physician 
Badri Rengarajan, MD  
 
+ 1-650-624-6946 
Fax 
+ 1-650-243-2312 
e-mail 
badri.rengarajan@actelion.com  
Observational Study Scientist 
Carol Zhao, MS   
 
+ 1-650-808-6616 
Fax 
+ 1-650-243-2312 
e-mail 
carol.zhao@actelion.com 
 
Clinical Study Manager 
Sandy Petersen-Stenger, MS  
 
+ 1-650-243-2359 
Fax 
+ 1-650-243-2312 
e-mail 
sandy.petersen@actelion.com  
Drug Safety Physician 
Roberto Cortes, MD 
 
+ 1-650-624-6945 
Fax 
+ 1-866-227-5886 
 
e-mail 
roberto.cortes@actelion.com 
Lead Principal Investigator 
 
Ellen Kim, MD (University of Pennsylvania) 
 
+ 1-215-662-6722 
e-mail  
ellen.kim@uphs.upenn.edu 
Study Biostatistician  
David Mink, MS (ICON Clinical Research) 
 
+ 1-415-371-2108 
e-mail 
david.mink@iconplc.com  

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SIGNATURE PAGE FOR ACTELION PHARMACEUTICALS US, INC. 
Hereinafter called Actelion 
TITLE: 
 
A prospective, observational, US-based study assessing 
outcomes, adverse events, treatment patterns, and quality of 
life in patients diagnosed with mycosis fungoides cutaneous 
T-cell lymphoma and treated with Valchlor® 
 
PROTOCOL NUMBER: AC-079A501 
AUTHORS: 
Badri Rengarajan, Director, Medical Affairs; 
Carol Zhao, Medical Affairs; 
Sandy Petersen-Stenger, Medical. 
 
 
 
REVIEWERS: 
Daniel Rosenberg, Senior Director, Epidemiology; 
Roberto Cortes, Drug Safety US; 
Laurence Oster, Senior Director, Pharmacovigilance 
Europe, EU QPPV; 
Lilla Di Scala, Senior Director, Biostatistics; 
Isabelle Leconte, Senior Director, CDDM; 
Frances Duffy-Warren, VP, Head Drug Regulatory US; 
Elke Hunsche, Head Global Market Access and Pricing 
Scientific Committee. 
 
 

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INVESTIGATOR/INSTITUTION SIGNATURE PAGE 
 
Disease area 
Mycosis fungoides cutaneous T-cell lymphoma 
Protocol number, acronym, study title, version 
Protocol AC-079A501, PROVe, A prospective, observational, US-based study assessing 
outcomes, treatment patterns, adverse events, and quality of life in patients diagnosed 
with mycosis fungoides cutaneous T-cell lymphoma and treated with Valchlor®. Final 
Version 3. 
 
I agree to the terms and conditions relating to this study as defined in this protocol and 
any other protocol-related documents. 
  
NAME 
(TITLE) 
 
CITY 
 
STATE  
 
DATE 
 
SIGNATURE 
 
 
 
 
 
 
 
 
 
 
 
 

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TABLE OF CONTENTS 
CONTACT DETAILS .........................................................................................................2 
LIST OF ABBREVIATIONS ..............................................................................................7 
PROTOCOL SYNOPSIS AC-079A501 ..............................................................................8 
PROTOCOL ......................................................................................................................17 
1 
AMENDMENTS AND UPDATES (PROTOCOL HISTORY) .................................17 
2 
STUDY MILESTONES ..............................................................................................17 
3 
RATIONALE AND BACKGROUND .......................................................................17 
4 
RESEARCH QUESTION AND OBJECTIVES .........................................................18 
5 
RESEARCH METHODS ............................................................................................19 
5.1 
Study design .................................................................................................19 
5.2 
Population (setting and study population) ....................................................19 
5.2.1 
Inclusion criteria ...............................................................................20 
5.2.2 
Exclusion criteria ..............................................................................20 
5.3 
Variables .......................................................................................................20 
5.3.1 
Outcome definition and measures .....................................................23 
5.3.2 
Exposure definition and measures ....................................................24 
5.3.3 
Covariate definition and measures ....................................................24 
5.4 
Data sources ..................................................................................................25 
5.5 
Study size ......................................................................................................25 
5.6 
Data management .........................................................................................26 
5.6.1 
Data collection ..................................................................................26 
5.7 
Data analysis .................................................................................................27 
5.7.1 
Analysis sets and groups ...................................................................27 
5.7.2 
Clinical characteristics ......................................................................27 
5.7.3 
Treatment and assessment patterns ...................................................27 
5.7.4 
Clinical outcomes .............................................................................28 
5.7.5 
Adverse events ..................................................................................28 
5.7.6 
Patient-completed questionnaires .....................................................28 
5.8 
Quality control ..............................................................................................29 
5.9 
Discussion of the study methods (including limitations) .............................29 
6 
PROTECTION OF HUMAN SUBJECTS AND ETHICAL APPROVAL ................30 

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6.1 
Institutional Review Board/Independent Ethics Committee approval .........30 
6.2 
Patient informed consent ..............................................................................30 
6.3 
Patient confidentiality ...................................................................................30 
7 
MANAGEMENT AND REPORTING OF ADVERSE EVENTS .............................31 
7.1 
Definitions ....................................................................................................31 
7.1.1 
Adverse event or adverse experience ................................................31 
7.1.2 
Serious adverse events ......................................................................32 
7.1.3 
Relationship to the use of Valchlor ...................................................33 
7.2 
Secondary exposures, AE and SAE reporting to Actelion US Drug 
Safety ............................................................................................................33 
7.2.1 
Follow-up of SAEs ...........................................................................34 
7.3 
Reporting of pregnancy ................................................................................34 
7.3.1 
Follow-up of pregnancy ....................................................................35 
7.4 
Reconciliation ...............................................................................................35 
8 
PLANS FOR DISSEMINATING AND COMMUNICATING STUDY RESULTS .35 
9 
DATA OWNERSHIP .................................................................................................35 
10 STUDY COMMITTEE(S) ..........................................................................................35 
11 REFERENCES ............................................................................................................36 
 
LIST OF TABLES 
Table 1 
eCRF Data Collection ............................................................................... 21 
 
LIST OF APPENDICES 
Appendix A 
Skindex-29 Questionnaire ......................................................................... 38 
Appendix B 
Skindex-29 Scoring algorithm ................................................................... 40 
Appendix C 
Visual Analog Scale for pruritus ............................................................... 41 
Appendix D 
Mycosis 
fungoides/Sézary 
syndrome-type 
cutaneous 
T-cell 
lymphoma .................................................................................................. 42 
Appendix E 
Mycosis 
fungoides/Sézary 
syndrome-type 
cutaneous 
T-cell 
lymphoma (CTCL QOL Scoring ............................................................... 43 
 
 

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LIST OF ABBREVIATIONS 
ADR  Adverse drug reaction 
AE  Adverse event 
BSA  Body surface area  
CAILS  Composite Assessment of Index Lesion Severity 
CTCL  Cutaneous T-cell lymphoma 
DCF  Data clarification form 
ECG  Electrocardiogram 
eCRF  Electronic Case Report Form 
EDC  Electronic data collection 
EOS  End of study 
FDA  Food and Drug Administration 
IEC  Independent Ethics Committee 
IRB  Institutional Review Board 
MCH  Mechlorethamine hydrochloride 
MCN  Manufacturer’s Control Number 
MedDRA  Medical Dictionary for Regulatory Activities 
MF-CTCL  Mycosis fungoides cutaneous T-cell lymphoma 
MF/SS-CTCL  Mycosis fungoides / Sézary syndrome-type cutaneous T-cell 
lymphoma  
mSWAT  Modified Severity Weighted Assessment Tool 
QOL  Quality of life 
SAE  Serious adverse event 
TNMB  Tumor-node-metastasis-blood 
VAS  Visual Analogue Scale 

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PROTOCOL SYNOPSIS AC-079A501 
This abstract is a stand-alone summary of the study protocol 
 
TITLE 
A 
PROspective, 
observational, 
US-based 
study 
assessing 
outcomes, adverse events, treatment patterns, and quality of life in 
patients diagnosed with mycosis fungoides cutaneous T-cell 
lymphoma (MF-CTCL) and treated with Valchlor® 
ACRONYM 
PROVe 
AUTHOR 
Badri Rengarajan, Actelion Pharmaceuticals US, Inc.  
RATIONALE 
AND 
BACKGROUND 
Due to the rarity of mycosis fungoides cutaneous T-cell lymphoma 
(MF-CTCL), there is a dearth of epidemiologic data detailing the 
clinical course of the disease and its response to treatment. While 
topical treatment with mechlorethamine hydrochloride (MCH) has 
been used since the 1950s, prospective data describing the clinical 
course of MF-CTCL is limited. Valchlor gel is a new formulation 
of MCH (or nitrogen mustard) that has been shown to be safe and 
effective in a clinical trial and has been approved for Stage I 
MF-CTCL patients  
Prospective data on Valchlor are currently only available from the 
pivotal, randomized, controlled clinical trial with strict inclusion 
and exclusion criteria. Understanding real-world patient safety and 
tolerability, as well as compliance and discontinuation rates, will 
assist with the management of Valchlor-treated MF-CTCL patients. 
This study will build and extend on the data from the Valchlor 
clinical development program, increase the understanding of the 
use of Valchlor in patients with MF-CTCL and, while limited to 
patients treated with Valchlor, will represent the largest prospective 
study with any given MF-CTCL treatment. 
This large, non-interventional study will provide information on the 
use of Valchlor in a ‘real-world’ clinical practice setting, and 
increase understanding of the management and outcomes of 
Valchlor-treated MF-CTCL patients. Data collected includes, but is 
not limited to: outcomes (e.g., clinical status and healthcare 
utilization [MF-CTCL-related hospitalizations and emergency room 
visits]), 
adverse 
events 
(AEs; 
e.g., 
dermatitis), 
clinical 
characteristics, and treatment patterns. Finally, this study will 
document disease burden (e.g., pruritus) of MF-CTCL in a real-life 

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setting by collecting patient-completed symptoms and quality of 
life (QOL) questionnaires for patients with skin disease. The QOL 
tools include VAS, Skindex-29 and a newly validated Mycosis 
fungoides / Sézary syndrome-type cutaneous T-cell lymphoma 
(MF/SS-CTCL) questionnaires. 
RESEARCH 
QUESTION AND 
OBJECTIVES 
The objectives of this study are: 
• To describe clinical characteristics of patients treated with 
Valchlor at enrollment and during the study observation period. 
• To describe treatment and response assessment patterns of 
patients with MF-CTCL treated with Valchlor at enrollment and 
during the study observation period. 
• To assess outcomes (e.g., clinical status and rates of healthcare 
utilization [MF-CTCL-related hospitalizations and emergency 
room visits]) in patients with MF-CTCL treated with Valchlor 
during the study observation period. 
• To collect and describe the incidence of AEs/serious adverse 
events (SAEs) in patients with MF-CTCL treated with Valchlor:  
o To estimate the incidence of dermatitis in patients using 
Valchlor during the study observation period. 
o To describe the management of dermatitis in patients 
using Valchlor at enrollment and during the study 
observation period.  
• To 
collect 
patient-completed 
symptoms 
and 
QOL 
questionnaires. 
STUDY DESIGN  
This is a multi-center, prospective, observational, US-based drug 
study. 
All consecutive MF-CTCL patients being treated with Valchlor will 
be invited to enroll in this study. 
Patients will undergo clinical assessments and receive standard 
medical care, as determined by the patients’ physician, in the 
real-world setting. With the exception of protocol-required 
patient-completed questionnaires for symptoms and QOL, there are 
no specific or mandated clinical assessments to be performed, and 
patients will not receive experimental intervention or treatment as a 
consequence of their participation in this study. 
Patients will be followed prospectively for a maximum of 2 years 

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from the date of signed informed consent (enrollment) until end of 
study, which includes withdrawal of consent, loss to follow-up, 
death, or study completion. Continuation in the study is not 
contingent on continuation of Valchlor.   
Patients currently enrolled and ongoing or newly enrolled will be 
offered to participate in the 24-month study of this protocol 
Amendment 1. Patients who enrolled under the original protocol 
(prior to Amendment 1) and are still ongoing in the study will be 
re-consented and to follow data collection specified in Table 1 of 
this protocol for a total of 24 months from original consent date.  
Patients who enrolled and have completed 1 year of the study under 
the original protocol will not be re-consented under Amendment 1.  
POPULATION 
(SETTING AND 
STUDY 
POPULATION) 
This study will enroll approximately 300 consecutive patients 
diagnosed with MF-CTCL and being treated with Valchlor, 
regardless of other MF-CTCL therapies received before or at 
enrollment. Patients will be enrolled from up to 50 participating 
centers/clinics in university-affiliated or community hospitals in the 
US. The participation of physicians and patients in the study is on a 
voluntary basis. 
Inclusion criteria: 
• All adult patients (≥ 18 years of age) diagnosed with MF-CTCL 
and being treated with Valchlor. This includes patients newly 
initiating Valchlor OR patients continuing treatment with 
Valchlor: 
o Patients newly initiating Valchlor are patients who have 
their first office visit after having initiated Valchlor.  
o Patients continuing treatment with Valchlor includes 
patients who are actively taking Valchlor on the day of 
enrollment. 
o Signed patient informed consent. 
Exclusion criteria: 
• None 
VARIABLES 
The variables of interest for this study includes clinical 
characteristics, treatment patterns, clinical status, as assessed by the 
physician (including the methods used in practice to assess clinical 
status), MF-CTCL-related healthcare utilization, as assessed by the 
physician (hospitalizations and emergency room visits), AEs and 

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SAEs, patient-completed symptoms and QOL questionnaires.  
Due to expected variability in practice patterns, no individual 
method of determining response is mandated, but assessments that 
do occur must be captured prospectively. Additionally, it is 
important to capture which methods were used pre-study, even 
where results may not be available, so that it is clear which 
assessments have a prior assessment that may have been considered 
in the physician’s evaluation of response. 
The specific outcomes and variables of interest for the study are: 
Clinical characteristics. 
Relevant medical history, including tumor-node-metastasis-blood 
(TNMB) classification. 
Use of prior MF-CTCL treatments. 
Treatment patterns, including prescribing information, for the 
treatment of MF-CTCL (including dosing regimens, titration 
schemes, duration of time from initiation to discontinuation of 
therapy, and drug holidays for Valchlor). 
Clinical status, as assessed by the physician (i.e., complete 
response, partial response, stable disease, progressive disease, 
relapse). 
Assessments (both whether or not the assessment was performed 
and the result of the assessment) used to define response, including, 
but not limited to: 
o Composite Assessment of Index Lesion Severity 
(CAILS). 
o Modified Severity Weighted Assessment Tool 
(mSWAT). 
o Physician’s Global Assessment. 
o Body Surface Area (BSA) of disease. 
Healthcare utilization related to MF-CTCL during the study period, 
as assessed by physician: 
o Number of MF-CTCL-related hospitalizations.  
o Number of MF-CTCL-related emergency room visits. 
AEs and SAEs (e.g., dermatitis) whether or not related to Valchlor. 

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Patient-completed questionnaires: 
o Visual Analogue Scale (VAS) for pruritus (scale 0–10).  
o Skindex-29. 
o MF/SS CTCL. 
DATA SOURCES 
Information will be collected per routine clinical practice. No 
specific clinical mandated assessments are to be performed as part 
of study participation. Patient-completed questionnaires, however, 
are a study requirement and will be collected at all clinic visits 
during the study observation period. These questionnaires will be 
administered on paper by study coordinators. Patient clinical data 
(non-patient-completed) will be collected from the patient’s 
medical records at enrollment and throughout the study period. All 
data will be entered into electronic Case Report Forms (eCRFs) at 
the time of collection or on a quarterly basis by study coordinators. 
AEs and SAEs whether or not related to Valchlor will be entered 
into the eCRF at every visit and upon notification by the patient 
(e.g., reported via phone call). 
For patients who do not have any visits, an affirmative response 
that the patient is still under care by the site will be requested 
quarterly, so that the absence of reported assessments can be 
interpreted the same as the actual absence of actual assessments. 
Pre-treatment information will be collected at the time of 
enrollment using the following variables: 
• Weight  
• TNMB classification  
• Use of Valchlor and other MF-CTCL treatments  
• Assessments used to define response (whether or not the 
assessment was ever performed, and regardless of whether the 
assessment was used to assess response for a previously treated 
patient or whether the assessment was performed for a 
comparison with future evaluations of response, and the results 
of the most recent assessments), including, but not limited to: 
o CAILS. 
o mSWAT. 
o Physician’s Global Assessment. 
o BSA of disease. 
In addition, the following information will be collected at the time 

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of enrollment:  
Patient demographics. 
Relevant medical history  
MF-CTCL TNMB classification. 
MF-CTCL histology. 
Use of Valchlor and other MF-CTCL treatments (including start 
and stop dates for each medication, and reasons for starting and 
stopping medications). 
Clinical status, as assessed by the physician (i.e., complete 
response, partial response, stable disease, progressive disease, 
relapse). 
Assessments (both whether or not the assessment was performed 
and the result of the assessment) used to assess response, including, 
but not limited to: 
o CAILS. 
o mSWAT. 
o Physician’s Global Assessment. 
o BSA of disease. 
All ongoing AEs and SAEs.  
Patient-completed questionnaires: 
o VAS for pruritus (scale 0–10). 
o Skindex-29.  
o MF/SS CTCL  
The following information will be collected at all clinic visits 
occurring during the observation period:  
MF-CTCL TNMB classification changes. 
Changes in relevant medical history. 
Use of Valchlor and other MF-CTCL treatments (including start 
and stop dates for each medication, and reasons for starting and 
stopping medications). 
Healthcare utilization related to MF-CTCL since the last study visit, 
as assessed by physician:  
o Number 
of 
MF-CTCL-related 
hospitalizations 

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(including length of stay and primary admission and 
discharge diagnoses). 
o Number of MF-CTCL-related emergency room visits. 
Clinical status, as assessed by physician (i.e., complete response, 
partial response, stable disease, progressive disease, relapse). 
Assessments (whether or not the assessment was performed and the 
result of the assessment) used to assess response, including, but not 
limited to: 
o CAILS. 
o mSWAT. 
o Physician’s Global Assessment. 
o BSA of disease. 
All AEs and SAEs (e.g., dermatitis) whether or not related to 
Valchlor, including reports of secondary exposures since last visit 
(while on Valchlor only). 
Patient-completed questionnaires:  
o VAS for pruritus (scale 0–10). 
o Skindex-29. 
o MF/SS-CTCL.  
Patients who enrolled under the original protocol and are still 
ongoing in the study will be contacted and informed consent will be 
obtained for the Amendment 1 protocol. Patients prospective data 
will be collected up to a total of 24 months from the original 
enrollment date. 
STUDY SIZE 
This study will enroll approximately 300 patients diagnosed with 
MF-CTCL who are being treated with Valchlor. 
A sample size of 270 evaluable patients is needed to estimate the 
percentage of patients with clinical status and the rate of 
hospitalizations, both with a margin of error of 6 percentage points. 
Assuming that up to 10% of patients may be non-evaluable due to 
incomplete follow-up, the sample size target for enrollment should 
be 300. It is projected that approximately 200 evaluable patients 
currently enrolled will be re-consented and newly enrolled will be 
consented under Amendment 1 for a 2-year follow-up. The margin 
of error for 200 evaluable patients will be 7%. 
Additionally, it is important to be able to estimate these same 

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percentages with a margin of error of 10 percentage points or less 
for key subgroups representing a third or more of the patient 
population (e.g., patients newly initiating Valchlor). A total of 
100 patients are required to achieve a margin of error of 
10 percentage points, so that a total enrollment of 300 patients is 
needed. 
DATA 
ANALYSIS 
Demographics and clinical characteristics will be summarized for 
all patients enrolled as well as grouped separately for patients 
continuing treatment with Valchlor versus patients who are newly 
initiating Valchlor. Use of prior treatments and past methods of 
assessing response will similarly be analyzed separately for the two 
groups. Treatments individually and in combination will be 
reported in percentage terms, by visit, stratified by newly initiating 
patients and continuing patients to identify treatment patterns. 
Analyses of response, based on a particular measure of response, 
will be limited to patients for whom that measure was reported.  
Therefore, patients with reported data will be summarized also for  
patients without any reported data with respect to demographics 
and pre-treatment clinical status to identify potential biases. Rates 
of on-study hospitalization and emergency room visits will be 
computed as the utilization counts divided by on-study days. The 
summary of response rates will be tabulated and the definition of 
response will be detailed in the Statistical Analysis Plan. 
The frequency and percentage of patients experiencing any AEs or 
SAEs will be summarized by system organ class and preferred 
MedDRA terminology. SAEs requiring hospitalizations and fatal 
SAEs will be displayed in patient listings. 
At each visit performed during the observation period (1st, 2nd, etc.), 
the VAS for pruritus, the three scales of the Skindex-29 and the 
MF/SS-CTCL questionnaires will be computed for patients who are 
and are not responders. These descriptive cross-sectional 
comparisons are intended to assess burden of disease in a 
Valchlor-treated cohort over time. 
MILESTONES 
First patient, first visit: Q4 2014 
Last patient, first visit: Q3 2017 
Last patient, last visit: Q3 2019 

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DATA 
OWNERSHIP 
Actelion Pharmaceuticals US, Inc. 
STUDY 
COMMITTEE(S) 
The Scientific Committee will consist of 4–6 key opinion leaders in 
the field of MF-CTCL who will provide scientific input on the 
MF-CTCL Study protocol and review study data. 

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PROTOCOL 
1 
AMENDMENTS AND UPDATES (PROTOCOL HISTORY) 
The protocol version 2, dated 12 November 2014, Appendix A, Appendix B and section 
5.7.6 for the Skindex-29 Questionnaire were updated. As these were administrative 
changes, no amendment was required; only a new protocol version number was issued. 
The protocol Amendment 1 will originate version 3 to extend ongoing patients and newly 
enrolled patients for a total of 24 months from consent date. An additional new quality of 
life assessment has been added. 
2 
STUDY MILESTONES 
This study will be conducted according to the following schedule: 
 
First patient, first visit: Q4 2014 
Last patient, first visit: Q3 2017 
Last patient, last visit: Q3 2019 
 
3 
RATIONALE AND BACKGROUND 
Background 
Mycosis fungoides cutaneous T-cell lymphoma (MF-CTCL) can present as patches, 
plaques, or tumors on the skin, often in areas of the body that are not usually exposed to 
the sun [Lymphoma.org 2011]. While MF-CTCL is rare, it is the most common type of 
cutaneous T-cell lymphoma (CTCL), accounting for 50–70% of all CTCLs [Imam 2013]. 
It is estimated that approximately 16,000 to 20,000 people in the US have the disease and 
that the 5-year incidence of MF-CTCL for all stages was 0.56 per 100,000 person-years 
between 2005–2009 [Korgavkar 2013, Leukemia & Lymphoma Society 2011]. The 
prevalence and incidence of MF-CTCL are likely to be under-reported due to 
nomenclature variances and because the symptoms and skin biopsy findings can appear 
similar to other types of skin conditions [Lymphoma.org 2011, Criscione 2007]. Patients 
frequently present with early stage disease typically associated with a favorable prognosis 
and survival of 10–35 years, but over 25% progress to advanced disease with a median 
survival of less than 4 years and just 13 months in those with nodal involvement 
[Scarisbrick 2014]. The most important clinical predictive factors for survival include 
patient age, T classification, and the presence of extracutaneous disease [Kim 2003a]. 
Although there is currently no cure for the disease, patients diagnosed at early stages of 
MF-CTCL (Stages IA, IB, or IIA) and receiving treatment for the disease have a median 
survival of 35.5, 21.5, and 15.8 years, respectively [Agar 2010]. Topical 
mechlorethamine hydrochloride (MCH or nitrogen mustard) has been used to treat 

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MF-CTCL since the 1950s [Lessin 2013]. The National Comprehensive Cancer Network 
[NCCN] Guidelines list topical MCH as a primary skin-directed treatment option for 
patients with MF-CTCL with limited/localized skin involvement or for generalized skin 
involvement [NCCN 2014]. Topical MCH has been available in aqueous and 
petrolatum-based forms. However, both forms have disadvantages that limit patient 
acceptance [Kim 2003b, Ramsay 1995].  
Valchlor, approved by the FDA in August 2013 for Stage IA and IB MF-CTCL, is a 
fast-drying, greaseless gel formulation that has been shown to be effective and safe in a 
clinical trial [Lessin 2013]. Two hundred sixty (260) patients in the Valchlor clinical trial 
(Study 201) received MF-CTCL specific treatment up to 12 months [Lessin 2013]. In the 
open-label, single group extension trial, 98 patients who completed Study 201 without a 
complete response received treatment with a formulation identical to Valchlor but 
containing MCH HCl 0.04% for 7 months. Currently, Valchlor is the only FDA-approved 
formulation of topical MCH, and one of two FDA-approved topical treatments for 
MF-CTCL [Kim 2014]. The results of the randomized, controlled, multi-center trial 
confirmed the non-inferiority of Valchlor in the treatment of MF-CTCL when compared 
to ointment-based MCH. 
Rationale 
Prospective data on Valchlor are currently only available from the pivotal, randomized, 
controlled clinical trial with strict inclusion and exclusion criteria. Understanding 
real-world patient safety and tolerability, as well as compliance and discontinuation rates, 
will assist with the future management of Valchlor-treated MF-CTCL patients. This study 
will build and extend on the data from the Valchlor clinical development program, 
increase the understanding of the use of Valchlor in patients with MF-CTCL and, while 
limited to patients treated with Valchlor, will represent the largest prospective study with 
any given MF-CTCL treatment. 
This large, non-interventional study will provide information on the use of Valchlor in a 
‘real-world’ clinical practice setting. This study will increase understanding of the 
management and outcomes of Valchlor-treated MF-CTCL patients by observing clinical 
characteristics, treatment patterns, outcomes (e.g., clinical status and healthcare 
utilization [MF-CTCL-related hospitalizations and emergency room visits]), and adverse 
events (AEs). Additionally, a better understanding of the disease burden of MF-CTCL 
will be gained by collecting patient-completed symptoms and quality of life (QOL) 
questionnaires.  
4 
RESEARCH QUESTION AND OBJECTIVES 
The objectives of the MF-CTCL prospective cohort study are: 

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• To describe clinical characteristics of patients treated with Valchlor at enrollment and 
during the study observation period. 
• To describe treatment and response assessment patterns of patients with MF-CTCL 
treated with Valchlor at enrollment and during the study observation period. 
• To assess outcomes (e.g., clinical status and rates of healthcare utilization 
[MF-CTCL-related hospitalizations and emergency room visits]) in patients with 
MF-CTCL treated with Valchlor during the study observation period. 
• To collect and describe the incidence of AEs/SAEs in patients with MF-CTCL treated 
with Valchlor:  
o To estimate the incidence of dermatitis in patients using Valchlor during the 
study observation period. 
o To describe the management of dermatitis in patients using Valchlor at 
enrollment and during the study observation period.  
• To collect patient-completed symptoms and QOL questionnaires. 
5 
RESEARCH METHODS 
5.1 Study design 
This is a multi-center, prospective, observational study of MF-CTCL patients who have 
newly initiated Valchlor or who are continuing treatment with Valchlor.  
Patients will be followed by their physician according to routine clinical practice. With 
the exception of protocol-required patient-completed questionnaires, there are no specific 
or mandated clinical assessments to be performed, and patients will not receive 
experimental intervention or treatment as a consequence of their participation in this 
study. The study protocol will not mandate any specific schedule of visits. Patients will 
undergo clinical assessments and receive standard medical care as determined by the 
patient’s physician in a real-world setting.  
Patients will be followed prospectively for a maximum of 2 years from the date of signed 
informed consent until end of study (EOS), which includes withdrawal of consent, loss to 
follow-up, death, or study completion. Continuation in the study is not contingent on 
continuation of Valchlor. 
The study will end after all enrolled patients have been followed for 2 years, or 
discontinued from the study for any reason. 
5.2 Population (setting and study population) 
This study will enroll approximately 300 MF-CTCL patients being treated (newly 
initiated or continuing treatment) with Valchlor [see Section 5.2.1 for details] from up to 
50 participating centers/clinics in university-affiliated or community hospitals. 

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Patients considered ‘being treated with Valchlor’ is comprised of two distinct groups: 
1) Patients newly initiating Valchlor. These are patients who are enrolled at their first 
office visit after having initiated Valchlor; 2) Patients continuing treatment with Valchlor. 
Patients must be actively taking Valchlor on the day of enrollment.  
All consecutive MF-CTCL patients being treated with Valchlor [see Section 5.2.1 for 
details] seen at the participating centers are eligible for enrollment in the study, regardless 
of other MF-CTCL therapies received before or at the time of enrollment. 
5.2.1 Inclusion criteria 
• All adult patients (≥ 18 years of age) diagnosed with MF-CTCL and being treated 
with Valchlor. This includes patients newly initiating Valchlor OR patients 
continuing treatment with Valchlor: 
o Patients newly initiating Valchlor are patients who have their first office visit 
after having initiated Valchlor.  
o Patients continuing treatment with Valchlor includes patients who are actively 
taking Valchlor on the day of enrollment. 
• Signed patient informed consent. 
5.2.2 Exclusion criteria 
• None. 
5.3 Variables  
The variables of interest for this study includes clinical characteristics at enrollment and 
during the study observation period, treatment patterns, clinical status, as assessed by the 
physician (including the methods used in practice to assess clinical status), 
MF-CTCL-related healthcare utilization, as assessed by the physician (hospitalizations 
and emergency room visits), AEs and SAEs, patient-completed symptoms and QOL 
questionnaires. 
Data elements to be collected from the patient’s medical record at study enrollment and at 
post-enrollment follow-up visits are shown in Table 1. Additionally, pre-treatment data 
collection using specific variables will be performed at enrollment. These variables 
include weight, tumor-node-metastasis-blood (TNMB) classification, use of Valchlor and 
other MF-CTCL treatments, and clinical status assessments. Drug holidays (temporary 
discontinuation) from Valchlor will be captured on the electronic Case Report Form 
(eCRF) by recording start and stop dates of all MF-CTCL treatments. Patient-completed 
questionnaires will be administered to patients who can read and understand English or 
Spanish and are able to self-complete the questionnaires based on the investigator’s 
assessment. Patients who are unable to read and understand English or Spanish will not 
be asked to complete the questionnaires and will be excluded from the study. 

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Table 1 
eCRF Data Collection 
Variables 
Enrollment visit 
Post-enrollment 
follow-up visits 
Informed Consent  
Demographics 
Age, gender, race/ethnicity, height 
Weight*  
X 
 
X 
X 
X1 
 
Relevant Medical History 
Age at diagnosis of MF-CTCL 
Date of MF-CTCL diagnosis 
Method of diagnosis 
MF-CTCL TNMB classification* 
MF-CTCL TNMB classification change 
MF-CTCL histology 
Malignancies and secondary malignancies 
Non-melanoma skin cancers 
Prior viral infections 
Atopic disorders 
Psoriasis 
Urticaria 
Mental disorders 
Family medical history 
Other relevant medical history 
Changes in medical history  
 
X 
X 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
X 
 
 
 
 
 
 
 
 
 
 
X 
Targeted MF-CTCL Treatments (Medication Use and 
Dosing)*† 
 
 
Valchlor 
X 
X 
Other MF-CTCL treatments 
X 
X 

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Variables 
Enrollment visit 
Post-enrollment 
follow-up visits 
Healthcare utilization related to MF-CTCL (as 
assessed by physician) 
Hospital admissions‡ 
Emergency room visits 
 
 
 
 
 
X 
 
X 
Clinical Status (as assessed by physician) 
X 
X 
Clinical Status Assessments* 
 
 
Composite Assessment of Index Lesion Severity  
X 
X 
Modified Severity Weighted Assessment Tool  
X 
X 
Physician’s Global Assessment 
X 
X 
Body Surface Area of disease 
X 
X 
Patient-Completed Questionnaires§ 
Visual Analogue Scale for pruritus 
Skindex-29 
Mycosis fungoides / Sézary syndrome-type cutaneous T-
cell lymphoma  
 
 
X 
X 
 
X 
 
 
X 
X 
 
X 
Safety Measures** 
AEs and SAEs  
 
X+ 
 
X 
Reports of secondary exposures 
 
X^ 
*Included in pre-treatment data collection, which is performed at enrollment. All data must be completed as 
available. 
†Includes collection of start and stop dates for each medication, including reasons for starting and stopping 
medications. Start and stop dates are also collected for Valchlor, including short gaps in treatment, such as 
drug holidays.  
‡For each hospital admission, length of stay and primary admission and discharge diagnoses will be 
collected. 
§To be completed by patients who can read and understand English and are able to self-complete the 
questionnaires based on the investigator’s assessment. Patients who are unable to self-complete the 
questionnaires will not be excluded from the study. 
**Safety measures, which include AEs and SAEs, will be collected from the informed consent date and 
prospectively at every visit during the study and up to 30-days post-discontinuation of Valchlor during the 
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New AEs and SAEs that occur more than 30 days after discontinuation of Valchlor will be reported to 
Actelion US Drug Safety if investigator feels there is a possible causal relationship with Valchlor. 
Investigators are also expected to assess patients for AEs and SAEs and follow the appropriate reporting 
procedures per Section 7. 
+All ongoing AEs and SAEs for patients being treated [see Section 5.2.1 for definition of “being treated”] 
with Valchlor. 
^All reports of secondary exposures, as available, will be counted on the eCRFs. Additional information 
regarding secondary exposures will be forwarded directly to Actelion US Drug Safety via the Secondary 
Exposure Form. Refer to Section 7.1.1 for definition of secondary exposure. 
AE = adverse event; eCRF = electronic Case Report Form; MF-CTCL = mycosis fungoides cutaneous 
T-cell lymphoma; SAE = serious adverse events; TNMB = tumor-node-metastasis-blood. 
1Re-consent at the first follow up visit for subjects who are currently active in the study prior to 
Amendment 1. 
5.3.1 Outcome definition and measures 
The clinical outcomes and variables of interest for this study are clinical characteristics, 
treatment patterns, clinical status, as assessed by the physician (and the methods used in 
practice to assess clinical status), MF-CTCL-related healthcare utilization, as assessed by 
the physician (hospitalizations and emergency room visits), AEs and SAEs (e.g., 
dermatitis), and patient-completed symptoms and QOL questionnaires. Due to expected 
variability in practice patterns, no individual method of determining response, either at 
follow-up or in tandem with a prior assessment, is mandated and a subjective categorical 
assessment of response will be included in addition to quantitative measures of response, 
but assessments that do occur must be captured prospectively. Additionally, it is 
important to capture which methods were used pre-study, even where results may not be 
available, so that it is clear which assessments have a prior assessment that may have 
been considered in the physician’s evaluation of response.  
For patients who do not have any assessments, an affirmative response that the patient is 
still under care by the site will be requested quarterly so that the absence of reported 
assessments can be interpreted the same as the absence of actual assessments. 
The following specific outcomes and variables of interest will be evaluated: 
Clinical characteristics at enrollment and during study observation period. 
Relevant medical history, including TNMB classification. 
Use of prior MF-CTCL treatments. 
Treatment patterns, including prescribing information, for the treatment of MF-CTCL 
(including dosing regimens, titration schemes, duration of time from initiation to 
discontinuation of therapy, and drug holidays for Valchlor). 
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stable disease, progressive disease, relapse). 
Assessments (both whether or not the assessment was performed and the result of the 
assessment) used to define response, including, but not limited to: 
o Composite Assessment of Index Lesion Severity. 
o Modified Severity Weighted Assessment Tool. 
o Physician’s Global Assessment. 
o Body Surface Area of disease. 
Healthcare utilization related to MF-CTCL during the study period, as assessed by 
physician: 
o Number of MF-CTCL-related hospitalizations.  
o Number of MF-CTCL-related emergency room visits.  
AEs and SAEs (e.g., dermatitis) whether or not related to Valchlor. 
Patient-completed questionnaires: 
o Visual Analogue Scale for pruritus (scale 0–10).  
o Skindex-29.  
o Mycosis fungoides / Sézary syndrome-type cutaneous T-cell lymphoma 
(MF/SS-CTCL). 
5.3.2 Exposure definition and measures 
Start of observation is defined as date of signed informed consent. 
End of observation is defined as EOS, including death, withdrawal of consent, loss to 
follow-up, or study completion, whichever occurs first.  
Total exposure time is defined as the date of first Valchlor exposure to the date of last 
Valchlor exposure. 
Total on-study Valchlor exposure time is defined as the first day of Valchlor use to last 
day of Valchlor use during the observation period. If the patient was using Valchlor at 
end of observation, the last on-study day of Valchlor use is equal to the end date of 
observation period. Drug holidays are excluded from the sum of exposure time. 
5.3.3 Covariate definition and measures 
The following covariates will be considered for analyses as well as for exploring their 
relationship to safety-related (e.g., dermatitis) outcomes: 
Age at enrollment, gender, race/ethnicity. 
MF-CTCL medical history: 
o Time since diagnosis and TNMB classification. 

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Medical history of previous and ongoing clinically significant underlying diseases and 
comorbid conditions. 
Prior exposure to Valchlor and other MF-CTCL specific therapies. 
5.4 Data sources  
Data elements to be collected include the following: patient demographics, personal and 
family medical history, including MF-CTCL diagnosis date and etiology, current and 
historical MF-CTCL treatments, clinical status (as assessed by the physician), and which 
assessments were used to define response, MF-CTCL-related healthcare utilization (as 
assessed by the physician), AEs and SAEs whether or not related to Valchlor, deaths, 
patient-completed symptoms and QOL questionnaires. Table 1 provides an overview of 
the variables collected at enrollment and during the observation period [see Section 5.3.2 
for definition]. For patients who do not have any visits, an affirmative response that the 
patient is still under care by the site will be requested quarterly, so that the absence of 
reported assessments can be interpreted the same as the absence of actual assessments. 
Since this is a real-world, observational study, there are a number of sources of bias that 
could potentially affect any conclusions that may be drawn. The methods used to 
minimize these sources of bias in this study are detailed below [see Section 5.9]. 
Therapies specific to MF-CTCL during the course of this study are under the direction of 
the participating physician and not the study protocol. As this study collects data on both 
incident patients and prevalent patients, it is necessary to include the date of diagnosis in 
the data collection forms, in order to minimize potential survivor bias effect. 
5.5 Study size 
This study will have an approximate enrollment of 300 patients diagnosed with 
MF-CTCL who are being treated with Valchlor (newly initiated or continuing treatment).  
A sample size of 270 evaluable patients is needed to estimate the percentage of patients 
with clinical status and the percentage of patients with hospitalizations, both with a 
margin of error of 6 percentage points. Assuming that up to 10% of patients may be 
non-evaluable due to incomplete follow-up, the sample size target for enrollment should 
be 300.  
It is projected that approximately 200 evaluable patients will be re-consented under 
Amendment 1. The margin of error for 200 evaluable patients will be 7%. 
Additionally, it is important to be able to estimate these same percentages with a margin 
of error of 10 percentage points or less for key subgroups representing a third or more of 
the patient population (e.g., patients newly initiating Valchlor). A total of 100 patients are 
required to achieve a margin of error of 10 percentage points per subgroup, so that a total 
enrollment of 300 patients is needed.  

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5.6 Data management  
5.6.1 Data collection 
Informed consent must be obtained prior to enrollment and data collection.  
Re-consent must be obtained for patients that were enrolled in the study prior to 
Amendment 1 and still ongoing to complete 24 months of data collection from the 
enrollment date. Instead of the 12 months initially planned.  
This study will collect data on MF-CTCL patients being treated with Valchlor [see 
Section 5.2.1 for inclusion criteria] under routine clinical care. This will include both data 
collected from assessments that are routinely performed for MF-CTCL patients being 
treated with Valchlor in clinical practice, and additional data from further assessments 
that may be performed. 
No specific clinically mandated assessments are to be performed as part of study 
participation. Patient-completed questionnaires, however, are a study requirement and 
will be collected at all clinic visits during the study observation period. These 
questionnaires will be administered on paper by study coordinators. Patient clinical data 
(non-patient-completed) will be collected from the patient’s medical records at 
enrollment and throughout the study period. All data will be entered into an eCRF at the 
time of collection or on a quarterly basis by study coordinators. AEs, reports of secondary 
exposure, and SAEs whether or not related to Valchlor will be entered into the eCRF at 
every visit and upon notification by the patient (e.g., reported via phone call). 
A secure, Internet-based electronic data collection (EDC) system will be used for data 
entry and hosting. An audit trail will be maintained by the EDC system for all data entries 
and changes, indicating what entries/changes have been made, who made them, and 
when. Data will be encrypted when transferred over the Internet and will be stored in a 
secure database protected from unauthorized access.  
All data collected will be de-identified before transmission to the central study 
coordinating center. Patient names will not be collected. Other patient identifiers will be 
collected in order to allow identification of study patients when SAEs are received by US 
Drug Safety outside of the study [see Section 7.4].  
All SAE data entered into the Adverse Event (AE)/Adverse Drug Reaction (ADR) Form 
and all reports of secondary exposure will be transferred electronically and/or by fax by 
the physician to Actelion US Drug Safety to be processed in the Actelion Drug Safety 
Database. In addition, all AE data, reports of secondary exposure, and SAE data will be 
entered into the study eCRF. 

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5.7 Data analysis  
5.7.1 Analysis sets and groups 
Demographics and clinical characteristics will be summarized for all enrolled patients 
and also grouped separately for patients continuing treatment with Valchlor versus 
patients who are newly initiating Valchlor. Use of prior treatments and past methods of 
assessing response will be similarly analyzed separately for the two groups. The methods 
used for assessment of response (i.e., assessed with that method versus not assessed with 
that method, either at follow-up alone or in tandem with a prior assessment) will be 
reported as cumulative percentages over time and also as a percentage of first visits, 
second visits, etc. To minimize the potential impact of missing data, analyses will focus 
on variables that are available for all patients, such as whether a particular type of 
assessment occurred, rather than focusing on the results of a particular assessment. 
Analyses of response, based on a particular measure of response, will be limited to 
patients for whom that measure was reported. Therefore, patients with reported data will 
be compared to patients without any reported data with respect to demographics and 
pre-treatment clinical status to identify potential biases. The summary of response rates 
will be tabulated and the definition of response will be detailed in the Statistical Analysis 
Plan. 
5.7.2 Clinical characteristics 
Analyses of prior treatment, assessments, and relevant medical history will be 
summarized for all patients enrolled and also grouped separately for patients newly 
initiating Valchlor versus patients continuing on Valchlor. Percentages will generally be 
computed as a function of non-missing data; however, if a large amount of data are 
missing for some historical data, a comparison of patients with missing and non-missing 
data may be necessary to ensure generalizability based on non-missing data alone. 
5.7.3 Treatment and assessment patterns 
Because relatively little prospectively collected data is available on MF-CTCL treatment 
patterns, preliminary analysis will make use of patient listings to develop categories of 
treatment patterns. Categories will be developed without cross-referencing outcomes data 
to minimize bias (i.e., the focus will be the presence of specific assessments, not the test 
value associated with the assessment). Treatments individually and in combination will 
be reported in percentage terms, by visit, stratified by newly initiating patients and 
continuing patients to identify treatment patterns. 
Duration of time from first post-initiation of Valchlor office visit to discontinuation of 
therapy will be assessed using Kaplan-Meier estimates of freedom from discontinuation. 
If short-term gaps in therapy are common, sensitivity analyses may be conducted in 
which these gaps do or do not count as treatment discontinuations, depending on the 
lengths of the gaps. These analyses will focus only on newly initiated patients. 

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5.7.4 Clinical outcomes 
The main clinical outcomes that will be analyzed are clinical response, as assessed by the 
physician, and MF-CTCL-related healthcare utilization, as assessed by the physician 
(e.g., hospitalizations and emergency room visits). Rates of on-study hospitalization and 
emergency room visits will be computed as the ratio of utilization counts divided by 
on-study days. Hospitalization-free survival will be computed using Kaplan-Meier 
estimates. 
Because a wide variation in assessments of clinical response is expected, the clinician’s 
subjective evaluation of response will be summarized in addition to reporting the 
proportion of responses among patients for whom specific tests were performed. The 
subjective response will be cross-classified by which assessments were used. The 
summary of response data is primarily a descriptive analysis to better understand how 
and when physicians define response, and it does not represent an estimate of the efficacy 
of any MF-CTCL treatment.  
5.7.5 Adverse events 
The frequency and percentage of patients experiencing any on-study AEs, SAEs, or death 
will be summarized by system organ class and preferred MedDRA terminology. 
Additional detail for AEs of special interest (e.g., dermatitis) will be summarized 
separately. SAEs will be displayed in patient listings, both including and excluding 
events that were not concurrent with Valchlor exposure. The definition of an on-study 
event is one that occurs on the day of enrollment or later, but before the date of study 
discontinuation or completion. 
5.7.6 Patient-completed questionnaires 
The Skindex-29 is a questionnaire that results in three scales: emotions, symptoms, and 
functioning [Chren 1996, Chren 1997a, Chren 1997b, Chren 2001]. Appendix A shows 
the items in the Skindex-29 questionnaire and Appendix B describes the scoring of 
individual items to the 3 scales. To compute the scale, each item is first transformed to a 
linear 0–100 scale where responses of “Never”, “Rarely”, “Sometimes”, “Often”, and 
“All the time” are converted to values of 0, 25, 50, 75, and 100, respectively. Next, the 
mean of the non-missing items for each scale is computed to derive a scale score. 
Although missing data for individual items is allowed, no scale score will be computed if 
more than quarter of the items in the scale are missing. 
The VAS for pruritus is a single value [Reich 2012] and is shown in Appendix C. 
The MF/SS-CTCL questionnaire contains 12 questions [PatientsLikeMe 2016] and is 
shown in Appendix D. 
At each study visit (1st, 2nd, etc.), the VAS for pruritus, the three scales of the Skindex-29 
and the MF/SS-CTCL will be computed for patients who are and are not responders, 

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based on clinician assessment, at the time of that visit. These descriptive cross-sectional 
comparisons are intended to assess burden of disease in a Valchlor-treated cohort over 
time. The data related to all three questionnaires (VAS, Skindex-29, MF/SS-CTCL) will 
be collected on the QOL questionnaires.  
5.8 Quality control  
The use of the EDC system will allow for on-line data quality checks upon data entry. 
The EDC system will have pre-programmed automatic edit checks for data 
inconsistencies at the time of data entry, and resulting queries will be addressed by the 
site. 
Site visits may be conducted by Actelion or by an authorized Actelion representative to 
inspect study data, patient medical records, and eCRFs. 
No source data verification is mandated. However, the data will be reviewed on an 
ongoing basis for implausible data points, missing data points, assessments not 
conducted, and/or incomplete information. This review may result in queries to the sites. 
Centralized remote monitoring of patient data will be performed via the EDC tool. This 
approach will allow proactive monitoring and querying of data on an ongoing and regular 
basis with concentrated efforts on key data points (e.g., hospitalization, death, Valchlor 
interruption and discontinuation, AEs and SAEs, and secondary exposures) to encourage 
low rates of missing information. Communication with investigators and study 
coordinators will emphasize the importance of data entry completion. Analytical methods 
described in Section 5.7 will be applied to assess the extent of the missing data and the 
impact on inferential estimates. 
5.9 Discussion of the study methods (including limitations) 
This study is an observational, prospective study examining MF-CTCL patients being 
treated with Valchlor in a real-world clinical setting. Valchlor treatment will be based on 
prescriber decision and will not be influenced by participation in this study. The patients 
will be followed by their physician according to routine clinical practice. The study 
protocol does not mandate any treatment, procedures or visits. Patient-completed 
questionnaires, however, are a study requirement and will be administered at all study 
visits for patients who can read and understand English, and are physically and mentally 
able to complete the questionnaires based on the investigator’s assessment. 
Any interpretation of observational study results should consider limitations to 
observational real-world study designs, including potential sources of selection or 
information biases, and confounding factors. 
To minimize selection bias, participating centers will offer enrollment in the study to all 
consecutive MF-CTCL patients currently treated with Valchlor [see Section 5.2.1 for 
inclusion criteria], regardless of other MF-CTCL specific therapy received before or at 

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the time of enrollment, or during the study. This study collects data on all users of 
Valchlor. At enrollment, a review of existing medical records will be performed to collect 
information as described per Table 1 (enrollment visit). Thereafter, all data will be 
collected during the observation period. 
Patients who are enrolled under the original protocol prior to protocol Amendment 1 and 
are still ongoing in the study will be re-consented under Amendment 1 and will continue 
to follow the schedule of Table 1 eCRF Data Collection specified in this protocol up to 
end of study (Month 24). Patients who enrolled and have completed 1 year of the study 
under the original protocol will not be offered to continue in the study and thus not 
re-consented under Amendment 1. 
To minimize the number of patients lost to follow-up, there will be regular contact with 
patients by the site, including 3 attempts to contact a patient before she/he is considered 
lost to follow-up. 
 
6 
PROTECTION OF HUMAN SUBJECTS AND ETHICAL APPROVAL 
6.1 Institutional Review Board/Independent Ethics Committee approval 
Depending on local regulations and the physician’s institutional policies, and in 
compliance with local law, the physician may have to submit the study protocol, the form 
for the Patient Informed Consent, and other relevant information to an Institutional 
Review Board (IRB) / Independent Ethics Committee (IEC), local health authorities, data 
protection agency or others. Approval from the IRB/IEC must be obtained before going 
through a consent procedure with the patient and before entering data into the database. 
IRB/IEC information/approval must be documented in a letter to the physician, clearly 
identifying the study, the documents reviewed, and the date of approval. 
6.2 Patient informed consent 
The participating physician commits to obtaining approval for the Patient Informed 
Consent Form from his/her respective Research Ethics Board, if required by applicable 
site policies, national privacy regulations and other state and local laws relating to 
medical information, prior to commencement of the project. Moreover, if required, the 
participating physician will obtain a signed Patient Informed Consent Form from each 
patient (or legal guardian) whose data will be included in the project.   
6.3 Patient confidentiality 
By signing the protocol, the participating physician commits to complying with all 
related applicable local laws and regulations, including but not limited to the regulations 
in 45 CFR parts 160 and 164 (protected health information), such regulations also known 
as the “HIPAA Privacy Regulations”. 

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7 
MANAGEMENT AND REPORTING OF ADVERSE EVENTS 
With reference to collecting, processing and expediting of AE reports, Actelion as the 
sponsor of this prospective non-interventional study complies with international and 
national pharmacovigilance regulations. 
7.1 Definitions 
7.1.1 Adverse event or adverse experience 
Any untoward medical occurrence in a patient administered a pharmaceutical product and 
which does not necessarily have a causal relationship with this treatment. An AE can 
therefore be any unfavorable and unintended sign (including an abnormal laboratory 
finding), symptom, or disease temporally associated with the use of a medicinal product, 
whether or not related to the medicinal product. 
AEs include: 
Recurrence or worsening of a pre-existing disease. 
Increase in frequency or intensity of symptoms of a pre-existing disease or medical 
condition. 
Continuous persistent disease or symptoms present at initiation of treatment or 
enrollment, which occurs first, that worsen following the start of the study. 
Lack of efficacy in the acute treatment of a life-threatening disease  Abnormal 
assessments, e.g., ECG, vital signs, or physical examination findings, if they represent a 
clinically significant finding that was not present at initiation of treatment or enrollment, 
which occurs first, or worsened during the course of the study or led to dose reduction, 
interruption or permanent discontinuation of Valchlor. 
Laboratory test abnormalities if they represent a clinically significant finding, 
symptomatic or not, which were not present at initiation of treatment or enrollment, 
which occurs first, or worsened during the course of the study or led to dose reduction, 
interruption or permanent discontinuation of Valchlor. 
In addition to AEs, exposure during pregnancy or lactation, product complaints, 
medication errors, overdose, misuse, abuse, occupational exposure and transmission of an 
infectious agent via Valchlor and the identification of a potential counterfeit medicinal 
product needs to be reported.  
Reports of off-label use without an associated AE collected within non-interventional 
studies shall be included in final study reports. 
Reports of inadvertent secondary exposure to the skin, mucous membranes, and eyes of 
individuals other than the patient being treated (e.g., patient’s caregiver or family 
member). 

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AEs do not include: 
Medical procedures such as surgery, endoscopy, tooth extraction. However, the event that 
led to the intervention is considered an AE. 
Situations in which no undesirable change occurred, such as hospitalization for cosmetic 
surgery or for social reasons. 
 
7.1.2 Serious adverse events 
An SAE is defined by the International Conference on Harmonisation guidelines as any 
AE fulfilling at least one of the following criteria: 
Fatal. 
Life-threatening: refers to an event in which the subject was at risk of death at the time of 
the event. It does not refer to an event that hypothetically might have caused death had it 
been more severe. 
Requiring inpatient hospitalization, or prolongation of existing hospitalization. 
Resulting in persistent or significant disability or incapacity. 
Congenital anomaly or birth defect. 
Medically significant: refers to important medical events that may not immediately result 
in death, be life-threatening, or require hospitalization but may be considered to be SAEs 
when, based upon appropriate medical judgment, they may jeopardize the subject, and 
may require medical or surgical intervention to prevent one of the outcomes listed in the 
definitions above. 
The following reasons for hospitalizations are exempted from being reported:  
Hospitalizations for cosmetic elective surgery, or social and/or convenience reasons. 
Hospitalization for pre-planned (prior to enrollment) standard monitoring of a 
pre-existing disease (present at enrollment) or medical condition that did not worsen, e.g., 
hospitalization for coronary angiography in a patient with stable angina pectoris. 
Hospitalizations for elective treatment of a pre-existing disease (present at enrollment) or 
medical condition that did not worsen, e.g., elective hip replacement for arthritis. 
New AEs and SAEs that occur > 30 days after the discontinuation of Valchlor unless 
there is a reasonable possibility of causal relationship to Valchlor. 
However, complications that occur during such hospitalizations are AEs or SAEs (for 
example, if a complication prolongs hospitalization) and must be reported as described 
below. 

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7.1.3 Relationship to the use of Valchlor 
Each AE must be assessed by the investigator as to whether or not there is a reasonable 
possibility of causal relationship to Valchlor, and reported as either related or unrelated. 
The determination of the likelihood that Valchlor caused the AE will be provided by an 
investigator who is a qualified physician. 
Related to Valchlor 
This category applies to any AE (whether serious or not) that appears to have a 
reasonable possibility of causal relationship to the use of Valchlor (i.e., a relationship 
cannot be ruled out). Guidelines to determine whether an event might be considered 
related include (but are not limited to) the following: 
There is temporal relationship between the occurrence of the event and the administration 
of Valchlor. 
The event abated (diminished) or disappeared when treatment with Valchlor was down-
titrated, interrupted, or discontinued.  
The event recurred when treatment was re-introduced.  
Unrelated to Valchlor 
This category applies to any AE (whether serious or not) that does not appear to have a 
reasonable relationship to the use of Valchlor (see above guidelines). Environmental 
factors such as clinical state and other treatments could equally have caused the event. 
7.2 Secondary exposures, AE and SAE reporting to Actelion US Drug Safety 
All reports of secondary exposures and all AEs will be recorded on the eCRFs and 
captured in the EDC clinical database. The AE data will be forwarded to Actelion US 
Drug Safety at predetermined intervals. 
All SAEs (including secondary exposures) which occur after initiation of Valchlor 
treatment until 30 days after Valchlor discontinuation must be collected, recorded and 
reported during the study. New AEs and SAEs that occur more than 30 days after the 
discontinuation of Valchlor will be reported to Actelion US Drug Safety if the 
investigator assesses that there is a possible causal relationship with Valchlor. The 
minimum information required for collecting and forwarding an SAE to Actelion US 
Drug Safety is the product name, patient identifier, reporter contact information, and the 
identified SAE. All SAEs must be recorded on an AE/ADR Form. Cases of secondary 
exposure must recorded on the Secondary Exposure Form and submitted directly to 
Actelion US Drug Safety. The AE/ADR Form will be pre-populated with the information 
already entered in the eCRF, and it is the physician’s responsibility to complete, print, 
and sign the form and forward to Actelion US Drug Safety directly, within 1 working day 

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of the investigator’s first knowledge of the event, by fax or using the drug safety email 
address:  
FAX: (866) 227-5886  
Actelion US Drug Safety: drugsafetyUS@actelion.com  
Actelion US Drug Safety will acknowledge the receipt of the AE/ADR form and provide 
a Manufacturer’s Control Number (MCN – a unique number assigned by Drug Safety’s 
database to the case) to the investigator and the Actelion Operations (Clinical Study 
Leader) within 48 hours. If no acknowledgement is received from Actelion US Drug 
Safety, the investigator must follow up immediately (or within 1 working day) with a 
phone call or email to Actelion US Drug Safety to ensure the AE/ADR Form has been 
received. 
7.2.1 Follow-up of SAEs 
Actelion US Drug safety will evaluate and process all safety reports received. If 
follow-up information is required, Actelion US Drug Safety will send a Data 
Clarification Form (DCF) directly to the investigator.  
Follow-up information about a previously reported SAE must be reported directly to 
Actelion US Drug Safety via the AE/ADR Form within 1 working day of the 
investigator’s first knowledge of the event.  
If the subject is hospitalized in a hospital other than the study site, it is the investigator’s 
responsibility to contact this hospital to obtain all SAE relevant information and 
documentation. 
SAEs still ongoing at EOS must be followed up for 30 days after Valchlor 
discontinuation or until resolution or stabilization, or until the event outcome is provided, 
e.g., fatal outcome. 
7.3 Reporting of pregnancy 
Any pregnancy occurring during the administration of Valchlor, including the 30 days 
following product discontinuation, must be reported within 24 hours or 1 working day of 
the investigator’s knowledge of the event.  
Pregnancies must be reported by the investigator on the Actelion Pregnancy Form, which 
is faxed or forwarded via the above-mentioned Actelion US Drug Safety email addresses 
within 24 hours of the investigator’s knowledge of the event.  
Any pregnancy diagnosed in the female partner of a male subject during treatment with 
Valchlor must be reported to Actelion US Drug Safety within 24 hours of the 
investigator’s knowledge of the event. 

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7.3.1 Follow-up of pregnancy 
If subsequent follow-up information is required, Actelion US Drug Safety will send a 
DCF directly to the investigator.  
Any pregnancy must be followed to its conclusion and its outcome must be reported to 
Actelion US Drug Safety directly.  
7.4 Reconciliation 
Actelion Operations (Clinical Study Leader) will keep a tracking log of all SAEs sent to 
Actelion US Drug Safety and the corresponding Actelion acknowledgement of receipt 
with Actelion MCN, so that reconciliation can be performed either periodically and/or at 
the end of the non-interventional study. 
8 
PLANS FOR DISSEMINATING AND COMMUNICATING STUDY 
RESULTS  
The Scientific Committee, in collaboration with Actelion, will publish data from this 
study at conferences and in journals when the sample size for analyses is sufficient. 
9 
DATA OWNERSHIP 
Actelion Pharmaceuticals US, Inc. owns the data. 
10 STUDY COMMITTEE(S) 
An independent Scientific Committee of 4–6 key opinion leaders in the field of 
MF-CTCL has been appointed and their responsibilities will provide scientific input on 
the MF-CTCL Study protocol and review of study data. 

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11 REFERENCES  
[Agar 2010] Agar NS, Wedgeworth E, Crichton S, et al. Survival outcomes and 
prognostic factors in mycosis fungoides/Sezary syndrome: validation of the revised 
International Society for Cutaneous Lymphomas/European Organisation for 
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2010;28(31):4730–39.  
[Chren 1996] Chren MM, Lasek RJ, Quinn LM, Mostow EN, Zyzanski SJ. Skindex, a 
quality-of-life measure for patients with skin disease: reliability, validity 
and responsiveness. J Invest Dermatol. 1996 Nov;107(5):707-13. 
[Chren 1997a] Chren MM, Lasek RJ, Quinn LM, Covinsky KE. Convergent and 
discriminant validity of a generic and a disease-specific instrument to measure 
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[Chren 1997b] Chren MM, Lasek RJ, Flock SA, Zyzanski SJ. Improved discriminative 
and evaluative capability of a refined version of skindex, a quality-of-life 
instrument for patients with skin diseases. Arch Dermatol. 1997;133:1433,1440. 
[Chren 2001] Chren MM, Lasek RJ, Sahay AP, Sands LP. Measurement of properties of 
skindex-16, a brief quality-of-life measure for patients with skin diseases. J Cutan 
Med Surg. 2001 Mar-Apr;5(2):105-10. Epub 2001 Mar 21.  
[Criscione 2007] Criscione VD, Weinstock MA. Incidence of cutaneous T-cell 
lymphoma in the United States, 1973–2002. Arch Dermatol. 2007;143(7):854–859.  
[Imam 2013] Imam MH, Shenoy PJ, Flowers CR, Phillips A, Lechowicz MJ. Incidence 
and survival patterns of cutaneous T-cell lymphomas in the United States. Leuk 
Lymphoma. 2013;54(4):752–9.  
[Kim 2003a] Kim YH, Liu HL, Mraz-Gernhard S, Varghese A, Hoppe RT. Long-term 
outcome of 525 patients with mycosis fungoides and Sezary syndrome: clinical 
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factors 
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risk 
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disease 
progression. 
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Jul 2003;139(7):857–66.  
[Kim 2003b] Kim YH, Martinez G, Varghese A, Hoppe RT. Topical nitrogen mustard in 
the management of mycosis fungoides: update of the Stanford experience. Arch 
Dermatol. 2003;139(2):165–73.  
[Kim 2014] Kim EE, Duvic M, Guitart J, Lessin S. Efficacy and safety of 
mechlorethamine (MCH) 0.04% gel in mycosis fungoides (MF) after treatment 
with topical MCH 0.02%. Presented at the American Society of Clinical Oncology 
(ASCO) 50th Annual Meeting. Chicago, IL. May 30–Jun 3 2014. Poster #9093. 
http://meetinglibrary.asco.org. 

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[Korgavkar 2013] Korgavkar K, Xiong M, Weinstock M. Changing incidence trends of 
cutaneous T-cell lymphoma. JAMA Dermatol. 2013;149(11):1295–1299.  
[Lessin 2013] Lessin SR, Duvic M, Guitart J, et al. Topical chemotherapy in cutaneous 
T-cell lymphoma: positive results of a randomized, controlled, multicenter trial 
testing the efficacy and safety of a novel mechlorethamine, 0.02%, gel in mycosis 
fungoides. JAMA Dermatol. 2013;149(1):25–32.  
[Leukemia & Lymphoma Society 2011] Leukemia & Lymphoma Society [Internet] 
Cutaneous T-Cell Lymphoma Facts 2011 [accessed 22 October 2014]. Available 
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cutaneoustcell.  
[Lymphoma.org 2011] Lymphoma Research Foundation. [Internet] Cutaneous T-cell 
lymphoma: Types of Cutaneous T-Cell Lymphoma [accessed 22 October 2014]. 
Available from: 
http://www.lymphoma.org/site/pp.asp?c=bkLTKaOQLmK8E&b=6300151.  
[NCCN 2014] National Comprehensive Cancer Network Clinical Practice Guidelines in 
Oncology: Non-Hodgkin’s Lymphomas. Version 4 .2014 [accessed on 
22 August 2014]. Available from: 
http://www.nccn.org/professionals/physician_gls/pdf/nhl.pdf.  
[PatientsLikeMe 2016] Creation of the Cutaneous T-cell Lymphoma-Quality of Life 
(CTCL-QoL) measure. Actelion Pharmaceuticals Ltd. Final Report. 31 May 2016.  
[Ramsay 1995] Ramsay DL, Meller JA, Zackheim HS. Topical treatment of early 
cutaneous T-cell lymphoma. Hematol Oncol Clin North Am. 1995;9(5):1031–56.  
[Reich 2012] Reich A, Heisig M, Phan NQ, et al. Visual analogue scale: evaluation of the 
instrument for the assessment of pruritus. Acta Derm Venereol. 2012;92(5): 
497–501.  
[Scarisbrick 2014] Scarisbrick JJ, Kim YH, Whittaker SJ, et al. Prognostic Factors, 
Prognostic Indices and Staging in Mycosis Fungoides and Sezary Syndrome: 
Where are we now? Br J Dermatol 2014 Jun;170(6):1226–36. 

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Appendix A 
Skindex-29 Questionnaire 
 

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Appendix B 
Skindex-29 Scoring algorithm 
 
 
 
 

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Appendix C 
Visual Analog Scale for pruritus  
 

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Appendix D 
Mycosis fungoides/Sézary syndrome-type cutaneous T-cell 
lymphoma 
 
 
 

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Appendix E 
Mycosis fungoides/Sézary syndrome-type cutaneous T-cell 
lymphoma (CTCL QOL Scoring)