An Observational Outcomes Study for Autologous Cell Therapy Among Patients With COPD and Interstitial Lung Disease

• Sponsor: Lung Institute (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03040674
• Collaborator: (none)
• Enrollment: 489
• Location: 1
• Actual Duration (Months): 47.9
• Patients Per Site Per Month: 10.2

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 same-day. Lung Institute's treatment is limited to self- funded patients with chronic lung disease - chronic obstructive pulmonary disease (COPD) and interstitial lung disease (ILD). The patient's cells and platelet rich plasma are collected through venous harvesting.

The hypothesized outcomes of Lung Institute therapy are safety and minimization of adverse events, a perceived improvement in the patient's lung condition (to be determined by their ability to be more physically active; walking greater distances with or without oxygen and improved quality of life scales), an improvement in the FEV1 among COPD patients, the ability to reduce their use of oxygen and possibly to stop it., the ability to function well without the use of rescue inhalers, reduction or ceasing 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 outcome data, Lung Institute aims to explore and describe the safety and efficacy of autologous cellular therapy 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, and to describe the anecdotal quality of life changes of patients following treatment using both quantitative and qualitative measures.

Study Design

• Study Type: Observational
• Actual Enrollment: 489 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: An Observational Outcomes Study for Autologous Cell Therapy Among Patients With COPD and Interstitial Lung Disease
• Actual Study Start Date: Jan 3, 2017
• Actual Primary Completion Date: Dec 31, 2020
• Actual Study Completion Date: Dec 31, 2020

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 Quality of Life (QOL) From Baseline [Measurements pre-treatment then at 3 and 6 months post-treatment]

   • Change from baseline in overall perceived quality of life over the course of a 6 month period as measured by the Clinical COPD Questionnaire (CCQ). The CCQ is a 10-item questionnaire scored as 0-6 for each item (0 being never and 6 being all the time with higher scores representing worse quality of life). The total score is 0-60. The change from baseline over the course of 6 months using the participant's self-reported responses to the CCQ. Higher total values on the CCQ demonstrate worsening quality of life.

2. Change in MMRC Scale From Baseline [Measurements pre-treatment then at 3 months post-treatment]

   • Change from baseline in Modified Medical Research Council (MMRC) dyspnea scale The change from baseline in perceived level of dyspnea over the course of 3 months using the participant's self-reported response to the MMRC. Possible scores are 0, 1, 2, 3 and 4 with higher scores representing severe loss of function due to breathing.

3. Change in FEV1 From Baseline [Measurements pre-treatment and at 3 months post-treatment]

   •Change from baseline in FEV1 (COPD patients only) as measured by pulmonary function testing/spirometry. The change from baseline in FEV1% predicted as measured by pulmonary function testing/spirometry over the course of 3 months.

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 nurse practitioner or physician of the Lung Institute who determines final eligibility for treatment. All eligible and thereby traded 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, Principal Investigator

Study Documents (Full-Text)

• Study Protocol - Dec 10, 2016
• Informed Consent Form - Mar 10, 2020

More Information

Additional Information:

• Clinic webpage

Publications

None provided.

• Responsible Party: Melissa Rubio, PhD, APRN, Principal Investigator, Lung Institute
• ClinicalTrials.gov Identifier: NCT03040674
• Other Study ID Numbers: LI002 Cohort A
• First Posted: Feb 2, 2017
• Last Update Posted: Apr 27, 2021
• Last Verified: Apr 1, 2021

• 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, Principal Investigator, Lung Institute: Shortness of breath; Breathlessness; Cellular therapy; Platelet rich plasma; Quality of life; Pulmonary function
• Additional relevant MeSH terms: Lung Diseases; Lung Diseases, Interstitial

Study Results

Participant Flow

Recruitment Details

Participants were recruited from the clinic site in Dallas, Texas. Participants were all diagnosed with either COPD or interstitial lung disease by their personal pulmonologist. Records review by provider including diagnosis, past medical history, medications, labs from within 3 months, lung function test results and the participants' ability to travel to and from the treatment site were assessed prior to approval. This study is IRB approved and each participant signed Informed Consent.

Pre-assignment Detail

All participants who started in the study were contacted at least twice at 3- and 6- months post-treatment by phone for quality of life follow-up. Participants were lost to follow-up at each time interval if they did not answer or return the call.

Arm/Group Title	Cell Therapy Treated
Arm/Group Description	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive autologous cellular therapy with cells harvested either from peripheral blood or from bone marrow plus peripheral blood. After harvest, cells are processed using minimal manipulation for platelet-rich plasma/ platelet concentrate (from peripheral blood) and/or mesenchymal stem cells (from bone marrow) and returned to the patient via peripheral circulation on the same day.
Period Title: Overall Study
STARTED	489
Participants Completing 3 Month Survey	351
Participants Completing 6 Month Survey	241
COMPLETED	241
NOT COMPLETED	248

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 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	489
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	125 25.6%
>=65 years	364 74.4%
Age (years) [Mean (Full Range) ]
Mean (Full Range) [years]	70.59
Sex: Female, Male (Count of Participants)
Female	173 35.4%
Male	316 64.6%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	4 0.8%
Native Hawaiian or Other Pacific Islander	3 0.6%
Black or African American	8 1.6%
White	461 94.3%
More than one race	1 0.2%
Unknown or Not Reported	12 2.5%
Chronic Lung Disease Diagnosis (Count of Participants)
Chronic Obstructive Pulmonary Disease (COPD)	357 73%
Interstitial Lung Disease (ILD)	132 27%

Outcome Measures

1. Primary Outcome

Title	Change in Quality of Life (QOL) From Baseline
Description	• Change from baseline in overall perceived quality of life over the course of a 6 month period as measured by the Clinical COPD Questionnaire (CCQ). The CCQ is a 10-item questionnaire scored as 0-6 for each item (0 being never and 6 being all the time with higher scores representing worse quality of life). The total score is 0-60. The change from baseline over the course of 6 months using the participant's self-reported responses to the CCQ. Higher total values on the CCQ demonstrate worsening quality of life.
Time Frame	Measurements pre-treatment then at 3 and 6 months post-treatment

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Cell Therapy Treated
Arm/Group Description	All patients/participants enrolled will undergo cell therapy Cell therapy: Each participant will receive autologous cellular therapy with cells harvested either from peripheral blood or from bone marrow plus peripheral blood. After harvest, cells are processed using minimal manipulation for platelet-rich plasma/ platelet concentrate (from peripheral blood) and/or mesenchymal stem cells (from bone marrow) and returned to the patient via peripheral circulation on the same day.
Measure Participants	489
Baseline CCQ Score	35.35 (10.56)
CCQ Score 3-Months Post-Treatment	27.96 (10.84)
CCQ Score 6 Months Post-Treatment	26.87 (10.84)

2. Primary Outcome

Title	Change in MMRC Scale From Baseline
Description	• Change from baseline in Modified Medical Research Council (MMRC) dyspnea scale The change from baseline in perceived level of dyspnea over the course of 3 months using the participant's self-reported response to the MMRC. Possible scores are 0, 1, 2, 3 and 4 with higher scores representing severe loss of function due to breathing.
Time Frame	Measurements pre-treatment then at 3 months post-treatment

Outcome Measure Data

Analysis Population Description

191 participants answered the MMRC questionnaire at baseline and again at 3-months post-treatment

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 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	191
Baseline	3 0.6%
3 Month Follow-Up	10 2%
Baseline	26 5.3%
3 Month Follow-Up	31 6.3%
Baseline	32 6.5%
3 Month Follow-Up	33 6.7%
Baseline	86 17.6%
3 Month Follow-Up	93 19%
Baseline	44 9%
3 Month Follow-Up	24 4.9%

3. Primary Outcome

Title	Change in FEV1 From Baseline
Description	•Change from baseline in FEV1 (COPD patients only) as measured by pulmonary function testing/spirometry. The change from baseline in FEV1% predicted as measured by pulmonary function testing/spirometry over the course of 3 months.
Time Frame	Measurements pre-treatment and at 3 months post-treatment

Outcome Measure Data

Analysis Population Description

175 participants with COPD had pulmonary function measurements taken at baseline and at 3-months post-treatment.

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 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	175
FEV1% Predicted at Baseline	34.2 (18.1)
FEV1% Predicted 3-Months Post-Treatment	35.8 (17.2)

Adverse Events

Time Frame	Adverse event data were collected for up to 1 year for each participant.
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 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	23/489 (4.7%)
Serious Adverse Events
	Cell Therapy Treated
	Affected / at Risk (%)	# Events
Total	0/489 (0%)
Other (Not Including Serious) Adverse Events
	Cell Therapy Treated
	Affected / at Risk (%)	# Events
Total	0/489 (0%)

Limitations/Caveats

This study was an observational study only with no control (placebo) group and was designed to examine the outcomes of participants following cellular therapy. This study makes no comparison to placebo or any other treatment. Participants incurred the cost of treatment and travel to the treatment center. This study and treatment protocol was approved by Advarra IRB for Human Subjects Protection.

More Information

Certain Agreements

Principal Investigators are NOT 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.