Self-Care for Head and Neck Cancer Survivors With Lymphedema and Fibrosis
Phase: NA
Conditions: Head and Neck Cancer, Lymphedema, Fibrosis
Sponsor: University of Pennsylvania

              
 
1 
 
 
 
 
 
Protocol & Statistical Analysis Plan 
 
 
 
 
 
Project Title: Self-Care for Head and Neck Cancer Survivors with Lymphedema and Fibrosis  
 
 
 
 
 
NCT 03030859 
 
 
 
 
 
Principal Investigator 
Jie Deng, PhD, RN, OCN, FAAN 
Associate Professor of Nursing 
University of Pennsylvania School of Nursing 
 
 
 
 
 
 
Funding Agency 
American Cancer Society 
 
 
Date: 8/10/2020 

 
1 
 
SECTION 1. SCIENTIFIC AIMS 
 
Aim 1: To determine the feasibility of a program of Lymphedema and Fibrosis – Self-Care Program 
(LEF-SCP) with or without follow-up to usual care for HNC survivors with LEF, specifically to: 1) obtain 
recruitment estimates and determine barriers to recruitment; 2) identify barriers to implementation; 3) 
assess safety; and 4) evaluate patient satisfaction.  
Aim 2: To determine if the LEF-SCP with or without follow-up enhances self-efficacy and adherence 
compared to usual care in HNC survivors with LEF.  
Aim 3: To determine the preliminary efficacy of the LEF-SCP with or without follow-up as compared to 
usual care for the following outcomes: 1) LEF progression; 2) symptom burden; and 3) functional 
status.  
 
SECTION 2. INCLUSION AND EXCLUSION CRITERIA 
 
Inclusion criteria: 1) post HNC primary treatment (chemotherapy and/or radiation and/or surgery); 2) no 
evidence of cancer (NED); 3) no more than 12 months after completion of initial lymphedema therapy 
for head and neck lymphedema; 4) > 18 years of age; 5) ability to understand English in order to 
complete questionnaires; 6) able to complete the onsite training and home self-care activities for LEF 
management; and 7) able to provide informed consent. Patients will be excluded if they have any of the 
following medical conditions that would prohibit the safe implementation of self-care of LEF: recurrent 
or metastatic cancer; any other active cancer; acute infection; congestive heart failure; renal failure; 
cardiac or pulmonary edema; sensitive carotid sinus; severe carotid blockage; uncontrolled 
hypertension; and venous thrombosis.  
 
SECTION 3. DESIGN/RECRUITMENT/ENROLLMENT/RANDOMIZATION 
 
We will conduct a prospective, randomized controlled trial to compare: Group 1-usual care, Group 2-
usual care plus LEF-SCP, and Group 3 - usual care plus LEF-SCP plus follow-up. Outcome measures 
include: 1) feasibility (barriers to implementation, safety, and satisfaction) of the proposed intervention 
[Aim 1]; 2) self-efficacy and adherence to self-care [Aim 2]; and 3) preliminary efficacy (LEF 
progression, symptom burden, and functional status) of the proposed intervention [Aim 3]. Our previous 
studies of HNC survivors with LEF support that 12-month follow-up assessments are possible. Thus, 
study follow-up assessments will take place at four points in time, 3-, 6-, 9-, and 12-month post-
intervention.  
 
Randomization: After completion of baseline measures, participants will be randomized via the use of 
a computer-generated, permuted block program, to one of three arms: Group 1 (usual care), Group 2 
(usual care plus LEF-SCP), and Group 3 (usual care plus LEF-SCP plus follow-up).  
 
SECTION 4. STUDY PROCEDURES 
 
Permission to conduct the proposed research will be obtained from the UPenn IRB. Written informed 
consent will be obtained from all participants. Potential participants will be approached and recruited at 
the Abramson Cancer Center. After signing informed consents participants will undergo baseline 
assessment measures. Upon completion of the baseline assessment, they will be informed of the 
subsequent study activities based on the groups they are assigned per randomization.  
 
SECTION 5. RISKS OF INVESTIGATIONAL AGENTS/DEVICES/SIDE EFFECTS 
Although this is an interventional study, there are no investigational devices or agents included in this 
study. Foreseeable physical, psychological, financial, legal, or other risks from study participation are 
believed to be minimal.  
 

 
2 
 
SECTION 6.  REPORTING OF UNANTICIPATED PROBLEMS INVOLVING RISK TO 
PARTICIPANTS OR OTHERS 
  
The PI Deng will review any adverse events, unanticipated problems, or side effects. PI Deng will report 
any adverse events, unanticipated problems, or side effects to: the UPenn IRB and the American 
Cancer Society.   
 
SECTION 7. STUDY WITHDRAWAL/DISCONTINUATION/ENDPOINTS 
 
Withdrawal and Discontinuation: Participants will be withdrawn from the study if they develop 
recurrent or metastatic cancer and any other active cancer.  
Endpoints: Final study visit 12 months after the study entry. 
 
SECTION 8. STATISTICAL ANALYSIS 
 
Statistical software (SPSS, STATA) will be used for the quantitative summarization of data collected in 
this study. Assumptions underlying each of the proposed statistical summaries and comparative 
analyses will be evaluated. Transformation of continuous data distributions will be completed as 
necessary to meet those assumptions. While this study is a preliminary study of efficacy and effect 
sizes, a maximum alpha level of 0.05 will be used for the statistical tests. 
 
Aim 1: Feasibility of the LEF-SCP. Descriptive statistical and graphical methods will summarize the 
rates of participation (recruited vs. consented), log data (e.g., sessions completed), completion of 
assessments throughout the study, adverse events (safety) and satisfaction data.    
Aim 2: Self-Efficacy and Self-Care Adherence. Descriptive statistical and graphical methods will be 
used to summarize and visually inspect the PMCSMS scores (self-efficacy), the number and types of 
self-care activities reported on the LEF Self-Care Checklist within the 3 study groups throughout the 
study period. Mixed effects generalized linear models that correct the standard errors for repeated 
assessments and incorporate appropriate distributional link functions (e.g., log-link for skewed 
distributions) will be used to generate estimates of the effect of the LEF-SCP on self-efficacy and rates 
of self-care.  
Aim 3: Preliminary Efficacy of the LEF-SCP. As with the previous aim, descriptive statistical and 
graphical methods will be used to summarize and visually inspect the measures of LEF, symptom 
burden, and functional status within the 3 study groups throughout the study period. Mixed effects 
generalized linear models with the appropriate link function for the nature of the specific outcome 
variable being analyzed will be used to generate estimates of the effect of the LEF-SCP on the 
progression of head and neck LEF, symptom burden, and function measures.  
 
SECTION 9. PRIVACY/CONFIDENTIALITY ISSUES 
 
Privacy:  The following procedure will be utilized to protect the privacy of the research participant. All 
data will be coded and filed without any name or other identifiable information. All files will be saved in 
both hard copies and one password protected safe electronic database by the researcher. All hard 
copies will be placed in a locking file cabinet. Only the study team members have access to the file 
cabinet and electronic database. All research team members will be certified with the IRB training and 
undergo HIPAA training. Privacy will be protected by conducting all study activities in a private room 
with a closed door. Careful training and supervision of all study staff will ensure procedures are carried 
out in accordance with established protocols.  
Confidentiality: All participants will be assigned a study ID number. Numbers will be assigned by 
trained study staff.  
 
SECTION 10. DATA AND SAFETY MONITORING PLAN 
 

 
3 
 
The data and safety monitoring plan will adhere to ACS policy guidance and standards set by the 
UPenn IRB. The PI will monitor all the study procedures, processes and data (including hard copy forms 
and electronic databases) monthly to ensure protocol compliance and data integrity and on an as-
needed basis if significant issues arise that impact the study participants or any study activities.