Trial Outcomes & Findings for Autologous Stem Cell Treatment for Chronic Lung Disease Study (NCT NCT03044431)

Last Updated: 2018-10-15

Results Overview

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

Recruitment status

COMPLETED

Target enrollment

207 participants

Primary outcome timeframe

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

Results posted on

2018-10-15

Participant Flow

All patients who underwent elective, investigational cell therapy were enrolled in the outcomes study.

No patients in the study received placebo. All patients received autologous, adult stem cell therapy.

Participant milestones

Participant milestones
Measure	Cell Therapy Treated 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 Study STARTED	207
Overall Study COMPLETED	148
Overall Study NOT COMPLETED	59

Reasons for withdrawal

Reasons for withdrawal
Measure	Cell Therapy Treated 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 Study Lost to Follow-up	59

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cell Therapy Treated n=207 Participants 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.
Age, Categorical <=18 years	0 Participants n=207 Participants
Age, Categorical Between 18 and 65 years	42 Participants n=207 Participants
Age, Categorical >=65 years	165 Participants n=207 Participants
Sex: Female, Male Female	95 Participants n=207 Participants
Sex: Female, Male Male	112 Participants n=207 Participants
Number of Participants with Chronic Obstructive Pulmonary Disease (COPD)	167 Participants n=207 Participants
Number of Participants with Interstitial Lung Disease (ILD)	40 Participants n=207 Participants
Treatment Type Venous Protocol	159 Participants n=207 Participants
Treatment Type Double Venous Protocol	26 Participants n=207 Participants
Treatment Type Bone Marrow Protocol	22 Participants n=207 Participants
Baseline FEV1 if participant has COPD	36.9 FEV1 Percentage (%) n=167 Participants • 167 patients in the overall sample were diagnosed with chronic obstructive pulmonary disease (COPD).
Baseline Clinical COPD Questionnaire Score All Participants	3.6 units on a scale n=207 Participants
COPD Participant Baseline QOL (quality of life) score based on the Clinical COPD Questionnaire (CCQ)	3.6 units on a scale n=167 Participants • 167 patients in the overall sample had a diagnosis of COPD.
ILD Participant Baseline QOL score based on Clinical COPD Questionnaire (CCQ)	3.3 units on a scale n=40 Participants • 40 patients in the sample were diagnosed with interstitial lung disease (ILD)

PRIMARY outcome

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

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

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

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=5 Participants 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.
Change in FEV1 From Baseline Among COPD Patients	12.8 percentage of change in FEV1 Interval 0.0 to 100.0

PRIMARY outcome

Timeframe: Measurements pre-treatment and then at 3 months post-treatment for all diagnoses

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

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).

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=148 Participants 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.
Number of Participants Reporting an Improvement in 3 Month-Post Treatment QOL Scores, All Diagnoses	121 Participants

PRIMARY outcome

Timeframe: Measurements pre-treatment and then at 6 months post-treatment among all diagnoses

Population: 124 patients completed a 6 month post-treatment QOL survey

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).

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=124 Participants 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.
Number of Participants Reporting an Improvement in 6 Month-Post Treatment QOL Scores, All Diagnoses	90 Participants

OTHER_PRE_SPECIFIED outcome

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

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

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).

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=120 Participants 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.
Number of Participants With COPD Reporting an Improvement in 3 Month-Post Treatment QOL Scores	102 Participants

OTHER_PRE_SPECIFIED outcome

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

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

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).

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=101 Participants 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.
Number of Participants With COPD Reporting an Improvement in 6 Month-Post Treatment QOL Scores	79 Participants

OTHER_PRE_SPECIFIED outcome

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

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

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).

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=28 Participants 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.
Number of Participants With Interstitial Lung Disease Reporting an Improvement in 3 Month-Post Treatment QOL Scores	20 Participants

OTHER_PRE_SPECIFIED outcome

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

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

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).

Outcome measures

Outcome measures
Measure	Cell Therapy Treated n=23 Participants 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.
Number of Participants With Interstitial Lung Disease Reporting an Improvement in 6 Month-Post Treatment QOL Scores	13 Participants

Adverse Events

Cell Therapy Treated

Serious events: 0 serious events

Other events: 10 other events

Deaths: 4 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Cell Therapy Treated n=207 participants at risk 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.
Surgical and medical procedures Dizziness	3.4% 7/207 • Number of events 7 • Adverse events monitoring and reporting was done over the entire length of the study, approximately 11 months from March 2016 through December 2016. 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.
Cardiac disorders Bradycardia, not procedure related	0.48% 1/207 • Number of events 1 • Adverse events monitoring and reporting was done over the entire length of the study, approximately 11 months from March 2016 through December 2016. 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.
Cardiac disorders Hypotension	0.97% 2/207 • Number of events 2 • Adverse events monitoring and reporting was done over the entire length of the study, approximately 11 months from March 2016 through December 2016. 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.

Additional Information

Melissa Rubio, PhD, APRN

Lung Institute

Phone: 214-504-2117

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place