Trial Outcomes & Findings for An Observational Outcomes Study for Autologous Cell Therapy Among Patients With COPD and Interstitial Lung Disease (NCT NCT03040674)

Last Updated: 2021-04-27

Results Overview

• 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.

Recruitment status

COMPLETED

Target enrollment

489 participants

Primary outcome timeframe

Measurements pre-treatment then at 3 and 6 months post-treatment

Results posted on

2021-04-27

Participant Flow

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.

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.

Participant milestones

Participant milestones
Measure	Cell Therapy Treated 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.
Overall Study STARTED	489
Overall Study Participants Completing 3 Month Survey	351
Overall Study Participants Completing 6 Month Survey	241
Overall Study COMPLETED	241
Overall Study NOT COMPLETED	248

Reasons for withdrawal

Reasons for withdrawal
Measure	Cell Therapy Treated 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.
Overall Study Lost to Follow-up	225
Overall Study Death	23

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cell Therapy Treated n=489 Participants 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
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	125 Participants n=5 Participants
Age, Categorical >=65 years	364 Participants n=5 Participants
Age, Continuous	70.59 years n=5 Participants
Sex: Female, Male Female	173 Participants n=5 Participants
Sex: Female, Male Male	316 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	4 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	3 Participants n=5 Participants
Race (NIH/OMB) Black or African American	8 Participants n=5 Participants
Race (NIH/OMB) White	461 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	12 Participants n=5 Participants
Chronic Lung Disease Diagnosis Chronic Obstructive Pulmonary Disease (COPD)	357 Participants n=5 Participants
Chronic Lung Disease Diagnosis Interstitial Lung Disease (ILD)	132 Participants n=5 Participants

PRIMARY outcome

Timeframe: Measurements pre-treatment then at 3 and 6 months post-treatment

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=489 Participants 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.
Change in Quality of Life (QOL) From Baseline Baseline CCQ Score	35.35 score on a scale Standard Deviation 10.56
Change in Quality of Life (QOL) From Baseline CCQ Score 3-Months Post-Treatment	27.96 score on a scale Standard Deviation 10.84
Change in Quality of Life (QOL) From Baseline CCQ Score 6 Months Post-Treatment	26.87 score on a scale Standard Deviation 10.84

PRIMARY outcome

Timeframe: Measurements pre-treatment then at 3 months post-treatment

Population: 191 participants answered the MMRC questionnaire at baseline and again 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.

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=191 Participants 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.
Change in MMRC Scale From Baseline MMRC Score 0 · Baseline	3 Participants
Change in MMRC Scale From Baseline MMRC Score 0 · 3 Month Follow-Up	10 Participants
Change in MMRC Scale From Baseline MMRC Score 1 · Baseline	26 Participants
Change in MMRC Scale From Baseline MMRC Score 1 · 3 Month Follow-Up	31 Participants
Change in MMRC Scale From Baseline MMRC Score 2 · Baseline	32 Participants
Change in MMRC Scale From Baseline MMRC Score 2 · 3 Month Follow-Up	33 Participants
Change in MMRC Scale From Baseline MMRC Score 3 · Baseline	86 Participants
Change in MMRC Scale From Baseline MMRC Score 3 · 3 Month Follow-Up	93 Participants
Change in MMRC Scale From Baseline MMRC Score 4 · Baseline	44 Participants
Change in MMRC Scale From Baseline MMRC Score 4 · 3 Month Follow-Up	24 Participants

PRIMARY outcome

Timeframe: Measurements pre-treatment and at 3 months post-treatment

Population: 175 participants with COPD had pulmonary function measurements taken at baseline 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.

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=175 Participants 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.
Change in FEV1 From Baseline FEV1% Predicted at Baseline	34.2 percentage of predicted Standard Deviation 18.1
Change in FEV1 From Baseline FEV1% Predicted 3-Months Post-Treatment	35.8 percentage of predicted Standard Deviation 17.2

Adverse Events

Cell Therapy Treated

Serious events: 0 serious events

Other events: 0 other events

Deaths: 23 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Melissa Rubio, PhD, APRN

Lung Health Institute

Phone: 2145042117

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place