COPD, Interstitial Lung Disease
Conditions
Keywords
Shortness of breath, Breathlessness, Cellular therapy, Platelet rich plasma, Quality of life, Pulmonary function
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.
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.
Interventions
PROCEDURECell 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
Sponsors
Lung Institute
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
Countries
United States
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 details
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.
Participants by arm
| Arm | Count |
|---|---|
| Cell Therapy Treated 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 | 489 |
| Total | 489 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 23 |
| Overall Study | Lost to Follow-up | 225 |
Baseline characteristics
| Characteristic | Cell Therapy Treated |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 364 Participants |
| Age, Categorical Between 18 and 65 years | 125 Participants |
| Age, Continuous | 70.59 years |
| Chronic Lung Disease Diagnosis Chronic Obstructive Pulmonary Disease (COPD) | 357 Participants |
| Chronic Lung Disease Diagnosis Interstitial Lung Disease (ILD) | 132 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 4 Participants |
| Race (NIH/OMB) Black or African American | 8 Participants |
| Race (NIH/OMB) More than one race | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 3 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 12 Participants |
| Race (NIH/OMB) White | 461 Participants |
| Sex: Female, Male Female | 173 Participants |
| Sex: Female, Male Male | 316 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 23 / 489 |
| other Total, other adverse events | 0 / 489 |
| serious Total, serious adverse events | 0 / 489 |
Outcome results
Primary
Change in FEV1 From Baseline
•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
Population: 175 participants with COPD had pulmonary function measurements taken at baseline and at 3-months post-treatment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cell Therapy Treated | Change in FEV1 From Baseline | FEV1% Predicted at Baseline | 34.2 percentage of predicted | Standard Deviation 18.1 |
| Cell Therapy Treated | Change in FEV1 From Baseline | FEV1% Predicted 3-Months Post-Treatment | 35.8 percentage of predicted | Standard Deviation 17.2 |
Primary
Change in MMRC Scale From Baseline
• 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
Population: 191 participants answered the MMRC questionnaire at baseline and again at 3-months post-treatment
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 0 | Baseline | 3 Participants |
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 0 | 3 Month Follow-Up | 10 Participants |
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 1 | Baseline | 26 Participants |
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 1 | 3 Month Follow-Up | 31 Participants |
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 2 | Baseline | 32 Participants |
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 2 | 3 Month Follow-Up | 33 Participants |
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 3 | Baseline | 86 Participants |
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 3 | 3 Month Follow-Up | 93 Participants |
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 4 | Baseline | 44 Participants |
| Cell Therapy Treated | Change in MMRC Scale From Baseline | MMRC Score 4 | 3 Month Follow-Up | 24 Participants |
Primary
Change in Quality of Life (QOL) From Baseline
• 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
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cell Therapy Treated | Change in Quality of Life (QOL) From Baseline | Baseline CCQ Score | 35.35 score on a scale | Standard Deviation 10.56 |
| Cell Therapy Treated | Change in Quality of Life (QOL) From Baseline | CCQ Score 3-Months Post-Treatment | 27.96 score on a scale | Standard Deviation 10.84 |
| Cell Therapy Treated | Change in Quality of Life (QOL) From Baseline | CCQ Score 6 Months Post-Treatment | 26.87 score on a scale | Standard Deviation 10.84 |