Autologous Stem Cell Treatment for Chronic Lung Disease Study

• ClinicalTrials.gov Identifier: NCT03044431
• Recruitment Status: Completed
• First Posted: February 7, 2017
• Results First Posted: October 25, 2017
• Last Update Posted: October 15, 2018
• Sponsor: Lung Institute
• Information provided by (Responsible Party): Melissa Rubio, PhD, APRN, Lung Institute

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Conditions: COPD; Interstitial Lung Disease
• Intervention: Procedure: Cell therapy
• Enrollment: 207

Participant Flow

Recruitment Details	All patients who underwent elective, investigational cell therapy were enrolled in the outcomes study.
Pre-assignment Details	No patients in the study received placebo. All patients received autologous, adult stem cell therapy.

Arm/Group Title	Cell Therapy Treated
Arm/Group Description	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
Period Title: Overall Study
Started	207
Completed	148
Not Completed	59
Reason Not Completed
Lost to Follow-up	59

Baseline Characteristics

Arm/Group Title		Cell Therapy Treated
Arm/Group Description		All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
Overall Number of Baseline Participants		207
Baseline Analysis Population Description		All patients who underwent cell therapy were included in the data analysis.
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	207 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	42 20.3%
	>=65 years	165 79.7%
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	207 participants
	Female	95 45.9%
	Male	112 54.1%
Race and Ethnicity Not Collected [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	0 participants
		[1] Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Number of Participants with Chronic Obstructive Pulmonary Disease (COPD); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	207 participants
		167 80.7%
Number of Participants with Interstitial Lung Disease (ILD); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	207 participants
		40 19.3%
Treatment Type; Measure Type: Count of Participants; Unit of measure: Participants
Venous Protocol	Number Analyzed	207 participants
		159 76.8%
Double Venous Protocol	Number Analyzed	207 participants
		26 12.6%
Bone Marrow Protocol	Number Analyzed	207 participants
		22 10.6%
Baseline FEV1 if participant has COPD [1]; Mean (Full Range); Unit of measure: FEV1 Percentage (%)
	Number Analyzed	167 participants
		36.9; (9 to 85)
		[1] Measure Analysis Population Description: 167 patients in the overall sample were diagnosed with chronic obstructive pulmonary disease (COPD).
Baseline Clinical COPD Questionnaire Score All Participants [1]; Mean (Full Range); Unit of measure: Units on a scale
	Number Analyzed	207 participants
		3.6; (0.8 to 6.0)
		[1] Measure Description: The Clinical COPD Questionnaire (CCQ) is a 10-item scale where the participant chooses responses on a 0-6 scale for each item. 0 represents no symptoms and 6 represents severe symptoms. The mean of the responses is the final score and ranges from 0-6, 0 is least severe symptoms and 6 is most severe symptoms.
COPD Participant Baseline QOL (quality of life) score based on the Clinical COPD Questionnaire (CCQ) [1] [2]; Mean (Full Range); Unit of measure: Units on a scale
	Number Analyzed	167 participants
		3.6; (1.0 to 6.0)
		[1] Measure Description: The Clinical COPD Questionnaire (CCQ) is a 10-item scale where the participant chooses responses on a 0-6 scale for each item. 0 represents no symptoms and 6 represents severe symptoms. The mean of the responses is the final score and ranges from 0-6, 0 is least severe symptoms and 6 is most severe symptoms.; [2] Measure Analysis Population Description: 167 patients in the overall sample had a diagnosis of COPD.
ILD Participant Baseline QOL score based on Clinical COPD Questionnaire (CCQ) [1] [2]; Mean (Inter-Quartile Range); Unit of measure: Units on a scale
	Number Analyzed	40 participants
		3.3; (0.8 to 5.6)
		[1] Measure Description: The Clinical COPD Questionnaire (CCQ) is a 10-item scale where the participant chooses responses on a 0-6 scale for each item. 0 represents no symptoms and 6 represents severe symptoms. The mean of the responses is the final score and ranges from 0-6, 0 is least severe symptoms and 6 is most severe symptoms.; [2] Measure Analysis Population Description: 40 patients in the sample were diagnosed with interstitial lung disease (ILD)

Outcome Measures

1. Primary Outcome

Title	Change in FEV1 From Baseline Among COPD Patients
Description	Change from baseline (among COPD patients only) as measured by pulmonary function testing/spirometry at baseline and again at 6 months post- treatment
Time Frame	Measurements pre-treatment and then at 6 months post- treatment

Outcome Measure Data

Analysis Population Description

All patients with COPD were asked to provide a 6 month post-treatment pulmonary function test. 5 participants completed and returned the test.

Arm/Group Title	Cell Therapy Treated
Arm/Group Description:	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
Overall Number of Participants Analyzed	5
Mean (Full Range); Unit of Measure: percentage of change in FEV1
	12.8; (0 to 100)

2. Primary Outcome

Title	Number of Participants Reporting an Improvement in 3 Month-Post Treatment QOL Scores, All Diagnoses
Description	Number of patients in the total sample who reported an improved perceived quality of life 3 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame	Measurements pre-treatment and then at 3 months post-treatment for all diagnoses

Outcome Measure Data

Analysis Population Description

148 of 207 enrolled participants completed the 3 month post-treatment QOL survey

Arm/Group Title	Cell Therapy Treated
Arm/Group Description:	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
Overall Number of Participants Analyzed	148
Measure Type: Count of Participants; Unit of Measure: Participants
	121 81.8%

3. Primary Outcome

Title	Number of Participants Reporting an Improvement in 6 Month-Post Treatment QOL Scores, All Diagnoses
Description	Number of patients in the total sample who reported an improved perceived quality of life 6 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame	Measurements pre-treatment and then at 6 months post-treatment among all diagnoses

Outcome Measure Data

Analysis Population Description

124 patients completed a 6 month post-treatment QOL survey

Arm/Group Title	Cell Therapy Treated
Arm/Group Description:	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
Overall Number of Participants Analyzed	124
Measure Type: Count of Participants; Unit of Measure: Participants
	90 72.6%

4. Other Pre-specified Outcome

Title	Number of Participants With COPD Reporting an Improvement in 3 Month-Post Treatment QOL Scores
Description	Number of patients with a diagnosis of COPD who reported an improved perceived quality of life 3 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame	Measurements for COPD pre-treatment and then at 3 months post-treatment

Outcome Measure Data

Analysis Population Description

120 patients with COPD completed a 3 month post-treatment QOL survey

Arm/Group Title	Cell Therapy Treated
Arm/Group Description:	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
Overall Number of Participants Analyzed	120
Measure Type: Count of Participants; Unit of Measure: Participants
	102 85.0%

5. Other Pre-specified Outcome

Title	Number of Participants With COPD Reporting an Improvement in 6 Month-Post Treatment QOL Scores
Description	Number of patients with a diagnosis of COPD who reported an improved perceived quality of life 6 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame	Measurements for COPD pre-treatment and then at 6 months post-treatment

Outcome Measure Data

Analysis Population Description

101 patients with COPD completed a 6 month post-treatment QOL survey

Arm/Group Title	Cell Therapy Treated
Arm/Group Description:	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
Overall Number of Participants Analyzed	101
Measure Type: Count of Participants; Unit of Measure: Participants
	79 78.2%

6. Other Pre-specified Outcome

Title	Number of Participants With Interstitial Lung Disease Reporting an Improvement in 3 Month-Post Treatment QOL Scores
Description	Number of patients with a diagnosis of Interstitial Lung Disease who reported an improved perceived quality of life 3 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame	Measurements for ILD pre-treatment and then at 3 months post-treatment

Outcome Measure Data

Analysis Population Description

28 patients with ILD completed a 3 month post-treatment QOL survey

Arm/Group Title	Cell Therapy Treated
Arm/Group Description:	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
Overall Number of Participants Analyzed	28
Measure Type: Count of Participants; Unit of Measure: Participants
	20 71.4%

7. Other Pre-specified Outcome

Title	Number of Participants With Interstitial Lung Disease Reporting an Improvement in 6 Month-Post Treatment QOL Scores
Description	Number of patients with a diagnosis of Interstitial Lung Disease who reported an improved perceived quality of life 6 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame	Measurements for ILD pre-treatment and then at 6 months post-treatment

Outcome Measure Data

Analysis Population Description

23 patients with ILD completed a 6 month post-treatment QOL survey

Arm/Group Title	Cell Therapy Treated
Arm/Group Description:	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
Overall Number of Participants Analyzed	23
Measure Type: Count of Participants; Unit of Measure: Participants
	13 56.5%

Adverse Events

Time Frame	Adverse events monitoring and reporting was done over the entire length of the study, approximately 11 months from March 2016 through December 2016.
Adverse Event Reporting Description	Any and all adverse events were reported to the Principal Investigator and reported to MaGil IRB and the supporting organization's quality and compliance monitor within 48 hours of the event.
Arm/Group Title	Cell Therapy Treated
Arm/Group Description	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.
All-Cause Mortality
	Cell Therapy Treated
	Affected / at Risk (%)
Total	4/207 (1.93%)
Serious Adverse Events
	Cell Therapy Treated
	Affected / at Risk (%)	# Events
Total	0/207 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0.2%
	Cell Therapy Treated
	Affected / at Risk (%)	# Events
Total	10/207 (4.83%)
Cardiac disorders
Bradycardia, not procedure related † 1 [1]	1/207 (0.48%)	1
Hypotension † 2 [2]	2/207 (0.97%)	2
Surgical and medical procedures
Dizziness † [3]	7/207 (3.38%)	7
1 Term from vocabulary, Bradycardia; 2 Term from vocabulary, Hypotension; † Indicates events were collected by systematic assessment; [1] Participant presented with pre-treatment significant bradycardia and was transported to the emergency department for evaluation. Treatment protocol stopped pending evaluation.; [2] Participants experienced low blood pressure following bone marrow harvesting of stem cells. Blood pressure was immediately corrected by the participant laying down and receiving intravenous fluids.; [3] Participant reported dizziness following collection of blood sample for venous cell therapy.

Limitations and Caveats

This study is limited by a small sample size for post-treatment pulmonary function test returns and the self-reporting of quality of life scores. The CCQ measure quantifiable and can show trends over time, but it remains a subjective measure.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: Melissa Rubio, PhD, APRN
• Organization: Lung Institute
• Phone: 214-504-2117
• EMail: pidallas@lunginstitute.com

• Responsible Party: Melissa Rubio, PhD, APRN, Lung Institute
• ClinicalTrials.gov Identifier: NCT03044431
• Other Study ID Numbers: LI001
• First Submitted: February 3, 2017
• First Posted: February 7, 2017
• Results First Submitted: June 27, 2017
• Results First Posted: October 25, 2017
• Last Update Posted: October 15, 2018