An Observational Outcomes Study for Autologous Cell Therapy Among Patients With COPD and Interstitial Lung Disease
Conditions: COPD, Interstitial Lung Disease
Sponsor: Lung Institute

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    RESEARCH PROTOCOL  
NCT03040674     
Protocol ID: LI002  
December 10, 2016 
  
 
An Observational Outcomes Study for Autologous Cell Therapy Among 
Patients With COPD and Interstitial Lung Disease 
 
Contents  
  
1.  Synopsis................................................................................................................................2 
2.  Abbreviations and Acronyms....................................................................................................2 
3.  Background ...........................................................................................................................2 
4.  Objectives .............................................................................................................................2 
5.  Study Methodology ................................................................................................................3 
6.  Study procedure.....................................................................................................................3 
7.  Data Management..................................................................................................................3 
8.  Adverse Event Reporting .........................................................................................................4 
9.  Ethical Issues .........................................................................................................................4 
10.  Finance and resource use ......................................................................................................4 
11.  Dissemination of Results and Publication policy ........................................................................4 
 
  
  
  
  
  
  

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1. Synopsis  
Management of COPD has primarily been with inhaled corticosteroids, oral steroids, lung reduction 
surgery and lung transplant but none of these treatments have proven ability to slow the progression of 
COPD over time.  Autologous, cellular therapy, using one’s own platelet rich plasma, is a novel 
approach to possibly help slow disease progression and possibly improve the patient’s quality of life.  
This study will descriptively explore the quality of life and lung function outcomes of participants at 3- 
and 6-months after cellular therapy.   
2. Abbreviations and Acronyms  
COPD= Chronic obstructive pulmonary disease  
PRP=Platelet rich plasma  
Background  
Conventional treatment for COPD usually involves inhaled bronchodilators, steroids, oxygen 
therapy and lung reduction surgery for the management of symptoms, although none of these 
modalities thwarts long-term progression of the disease. Aside from lung transplantation, 
there is no cure for the disease, the availability of donor lungs is scarce, and the long-term 
need for anti-rejection medication and monitoring can be overwhelming.  Recent advances in 
the field of regenerative medicine in lung disease have demonstrated that autologous cellular 
therapy may reduce inflammation and aid in the maintenance and repair of damaged lung 
tissue,   As a result, some patients have elected to undergo autologous cellular therapy 
utilizing their body’s own cells in an effort to control their chronic lung disease.    
  
In the case of this study, cells were harvested from the patient by either the bone marrow or 
peripheral blood, isolated and concentrated using centrifugation, and then returned to the 
patient same-day via the peripheral circulation.  As circulation occurs, the concentrated cells 
enter the right heart and are then disseminated into the lungs, becoming trapped in the 
lung’s microcirculation.  Here, the cells begin to produce bioactive factors such as cytokines 
and anti-inflammatory mediators.  Several growth factors are released by activated platelets 
becoming honing cells for healing within the tissue. The exact long-term mechanism of action 
of stem cells and PRP in the lungs remains under investigation.  But because of the minimal 
cell manipulation and the autologous nature of the cells, the risk of adverse events is low.   
  
3. Objectives   
The primary objective of this study is to descriptively examine the outcomes on lung function 
and perceived quality of life at 3 months and 6 months following autologous cellular therapy 
treatment.   

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4. Study Methodology   
This is a descriptive, observational study only and all participants received cellular therapy. 
This is not an experimental design and there is no placebo/control group.   
Study population  
 All persons undergoing treatment at the Lung Health Institute facility in Dallas, Texas 
from March 2016 through October 2020 will participate in this study.   
5. Study procedure   
 Potential participants will be presented with study information  
 Each potential participant will have a consultation with a medical provider prior to 
enrollment to determine eligibility for treatment.   
  Participants will be presented with an informed consent by study staff.   
  Baseline spirometry with a focus on FEV1 volume and FEV1% predicted 
will be collected on each participant. The CCQ (Clinical COPD 
Questionnaire) will be collected on each participant at baseline to assess 
quality of life.    
  Participants will be followed at 3 months and 6 months. At both follow-up 
timepoints, each participant will complete the CCQ quality of life 
questionnaire. Participants will be asked to return after 3 months for follow-
up spirometry. All data is documented in the participant’s secure electronic 
medical record.   
  The procedure for obtaining and processing the venous blood will be the 
same for all participants. Whole blood is drawn from the peripheral 
circulation, processed with specialized centrifugation using commercial 
equipment, and a final platelet rich plasma/platelet concentrate product 
prepared.    
  Each participant will receive a reinfusion of platelet rich plasma/platelet 
concentrate on each of two, consecutive treatment days.   
  
 Study timelines:  The study will close once 12-month post-treatment follow-up has been 
completed for each participant.   
  
6. Data Management   
 Data will be stored in a password-protected database and secure, encrypted electronic 
medical record.  
 All patient identifiers will be removed for data analysis and dissemination of findings.  

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 Demographics, quality of life data and spirometry data will be summarized, and pre-and 
post- treatment quality of life and spirometry data will be compared in descriptive format.   
Tests of statistical significance will be performed on the difference.   
7. Adverse Event Reporting  
• Any events that are not anticipated are reported directly to the Principal 
Investigator and to the IRB within 24 hours of occurrence.   
8. Ethical Issues  
  There are no dual relationships, coercion or inducement to disclose  
  There are no non-negligible risks or burdens to participants anticipated  
9. Finance and resource use  
Participants will not be paid or reimbursed anything for their participation in the study.  Their 
participation is voluntary, and they may withdraw their participation at any time.   
10. 
Dissemination of Results and Publication policy  
The study findings will be disseminated to any interested party as well as potentially prepared 
for publication. Other interested parties may include healthcare professionals and policymakers 
in the fields of primary care, pulmonology and regenerative medicine.