Autologous Stem Cell Treatment for Chronic Lung Disease Study

• Sponsor: Lung Institute (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03044431
• Collaborator: (none)
• Enrollment: 207
• Location: 1
• Actual Duration (Months): 14.3
• Patients Per Site Per Month: 14.5

Study Details

Study Description

Brief Summary

The purpose of the Lung Institute is to collect and isolate a patient's own cells and platelet rich plasma (PRP) and deliver the product back to the patient the same day. Lung Institute's treatment is limited to self-funded patients with chronic lung disease- chronic obstructive pulmonary disease (COPD) and restrictive lung diseases such as pulmonary fibrosis (PF) and interstitial lung disease (ILD). The patient's cells and platelet rich plasma are harvested through venous or bone marrow collection techniques.

The hypothesized outcomes of therapy are safety and minimization of adverse events, a perceived improvement in the patient's lung condition (to be determined by their perceived quality of life), an improvement in the FEV1 among COPD patients, the ability to reduce supplemental oxygen use, the ability to function well without the use of rescue inhalers, reduction of secondary pulmonary infections, reduction in emergency room visits and exacerbations related to their disease.

Condition or Disease	Intervention/Treatment	Phase
COPD; Interstitial Lung Disease	Procedure: Cell therapy

Detailed Description

Through the collection of outcomes data, Lung Institute aims to explore and describe the safety and efficacy of autologous stem cell treatment for chronic lung disease with dissemination to the public and to the medical community for the advancement of regenerative medicine. The study aims to confirm the safety of autologous cell therapy, explore the effect of autologous cell therapy treatment on pulmonary function over time, and to describe the anecdotal quality of life changes of patients following treatment using quantitative and qualitative measures.

Study Design

• Study Type: Observational
• Actual Enrollment: 207 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Autologous Stem Cell Treatment for Chronic Lung Disease Study
• Actual Study Start Date: Apr 1, 2016
• Actual Primary Completion Date: Jun 1, 2017
• Actual Study Completion Date: Jun 9, 2017

Arms and Interventions

Arm	Intervention/Treatment
Cell therapy treated All patients/participants enrolled will undergo cell therapy	Procedure: 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.

Outcome Measures

Primary Outcome Measures

1. Change in FEV1 From Baseline Among COPD Patients [Measurements pre-treatment and then at 6 months post- treatment]

   Change from baseline (among COPD patients only) as measured by pulmonary function testing/spirometry at baseline and again at 6 months post- treatment

2. Number of Participants Reporting an Improvement in 3 Month-Post Treatment QOL Scores, All Diagnoses [Measurements pre-treatment and then at 3 months post-treatment for all diagnoses]

   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).

3. Number of Participants Reporting an Improvement in 6 Month-Post Treatment QOL Scores, All Diagnoses [Measurements pre-treatment and then at 6 months post-treatment among all diagnoses]

   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).

Other Outcome Measures

1. Number of Participants With COPD Reporting an Improvement in 3 Month-Post Treatment QOL Scores [Measurements for COPD pre-treatment and then at 3 months post-treatment]

   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).

2. Number of Participants With COPD Reporting an Improvement in 6 Month-Post Treatment QOL Scores [Measurements for COPD pre-treatment and then at 6 months post-treatment]

   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).

3. Number of Participants With Interstitial Lung Disease Reporting an Improvement in 3 Month-Post Treatment QOL Scores [Measurements for ILD pre-treatment and then at 3 months post-treatment]

   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).

4. Number of Participants With Interstitial Lung Disease Reporting an Improvement in 6 Month-Post Treatment QOL Scores [Measurements for ILD pre-treatment and then at 6 months post-treatment]

   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).

Eligibility Criteria

Criteria

• Ages Eligible for Study: 16 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Patients are included in treatment by self-referral and after consultation with a designated patient coordinator who determines initial eligibility, and then by the clinic nurse practitioner or physician of Lung Institute who determines final eligibility for inclusion. All eligible treated patients are eligible for the study.

Exclusion Criteria:

• Patients who are unable to provide informed consent, pregnant patients, prisoners, non-English speakers, patients with a history of cancer within the past 5 years, patients with active tuberculosis or pneumonia. In addition, patients on prescribed blood thinners, with a history of breast or prostate cancer of any time frame or with a history of osteoporosis are excluded from the bone marrow harvest option.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Lung Institute Dallas	Dallas	Texas	United States	75231

Sponsors and Collaborators

• Lung Institute

Investigators

• Principal Investigator: Melissa Rubio, PhD, APRN, Lung Institute Dallas

Study Documents (Full-Text)

• Study Protocol, Statistical Analysis Plan, and Informed Consent Form - Sep 22, 2016

More Information

Additional Information:

• Clinic webpage

Publications

None provided.

• Responsible Party: Melissa Rubio, PhD, APRN, PhD, APRN, Lung Institute
• ClinicalTrials.gov Identifier: NCT03044431
• Other Study ID Numbers: LI001
• First Posted: Feb 7, 2017
• Last Update Posted: Oct 15, 2018
• Last Verified: Oct 1, 2018

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Melissa Rubio, PhD, APRN, PhD, APRN, Lung Institute: Shortness of breath; Breathlessness; Stem cells; Platelet rich plasma; Quality of life; Pulmonary Function
• Additional relevant MeSH terms: Lung Diseases; Lung Diseases, Interstitial

Study Results

Participant Flow

Recruitment Details	All patients who underwent elective, investigational cell therapy were enrolled in the outcomes study.
Pre-assignment Detail	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

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 Participants	207
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	42 20.3%
>=65 years	165 79.7%
Sex: Female, Male (Count of Participants)
Female	95 45.9%
Male	112 54.1%
Race and Ethnicity Not Collected (Count of Participants)
Number of Participants with Chronic Obstructive Pulmonary Disease (COPD) (Count of Participants)
Count of Participants [Participants]	167 80.7%
Number of Participants with Interstitial Lung Disease (ILD) (Count of Participants)
Count of Participants [Participants]	40 19.3%
Treatment Type (Count of Participants)
Venous Protocol	159 76.8%
Double Venous Protocol	26 12.6%
Bone Marrow Protocol	22 10.6%
Baseline FEV1 if participant has COPD (FEV1 Percentage (%)) [Mean (Full Range) ]
Mean (Full Range) [FEV1 Percentage (%)]	36.9
Baseline Clinical COPD Questionnaire Score All Participants (units on a scale) [Mean (Full Range) ]
Mean (Full Range) [units on a scale]	3.6
COPD Participant Baseline QOL (quality of life) score based on the Clinical COPD Questionnaire (CCQ) (units on a scale) [Mean (Full Range) ]
Mean (Full Range) [units on a scale]	3.6
ILD Participant Baseline QOL score based on Clinical COPD Questionnaire (CCQ) (units on a scale) [Mean (Inter-Quartile Range) ]
Mean (Inter-Quartile Range) [units on a scale]	3.3

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.
Measure Participants	5
Mean (Full Range) [percentage of change in FEV1]	12.8

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.
Measure Participants	148
Count of Participants [Participants]	121 58.5%

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.
Measure Participants	124
Count of Participants [Participants]	90 43.5%

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.
Measure Participants	120
Count of Participants [Participants]	102 49.3%

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.
Measure Participants	101
Count of Participants [Participants]	79 38.2%

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.
Measure Participants	28
Count of Participants [Participants]	20 9.7%

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.
Measure Participants	23
Count of Participants [Participants]	13 6.3%

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 (%)	# Events
Total	4/207 (1.9%)
Serious Adverse Events
	Cell Therapy Treated
	Affected / at Risk (%)	# Events
Total	0/207 (0%)
Other (Not Including Serious) Adverse Events
	Cell Therapy Treated
	Affected / at Risk (%)	# Events
Total	10/207 (4.8%)
Cardiac disorders
Bradycardia, not procedure related	1/207 (0.5%)	1
Hypotension	2/207 (1%)	2
Surgical and medical procedures
Dizziness	7/207 (3.4%)	7

Limitations/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.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.