Back to resultsPROCHYMAL™ (Human Adult Stem Cells) for the Treatment of Moderate to Severe Chronic Obstructive Pulmonary Disease (COPD)
Primary Purpose
Pulmonary Disease, Chronic Obstructive, Pulmonary Emphysema, Chronic Bronchitis
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Prochymal™
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Pulmonary Disease, Chronic Obstructive focused on measuring COPD, Airflow Obstruction, Chronic, Chronic Airflow Obstruction, Chronic Obstructive Airway Disease, Chronic Obstructive Lung Disease, Chronic Obstructive Pulmonary Disease, Pulmonary Emphysema, Chronic Bronchitis, Mesenchymal Stem Cells (MSCs), Adult Human Stem Cells, Osiris, Prochymal™
Eligibility Criteria
Inclusion Criteria:
- Participant must have a diagnosis of moderate or severe COPD.
- Participant must have a post-bronchodilator FEV1/forced vital capacity (FVC) ratio < 0.7.
- Participant must have a post-bronchodilator FEV1 % predicted value ≥ 30% and < 70%.
- Participant must be between 40 and 80 years of age, of either sex, and of any race.
- Participant must be a current or ex-smoker, with a cigarette smoking history of ≥ 10 years or > 10 pack-years.
Exclusion Criteria:
- Participant has been diagnosed with asthma or other clinically relevant lung disease other than COPD (e.g. restrictive lung diseases, sarcoidosis, tuberculosis, idiopathic pulmonary fibrosis, bronchiectasis, or lung cancer).
- Participant has been diagnosed with α1-antitrypsin deficiency.
- Participant has a body mass greater than 150 kg (330 lb) or less than 40 kg (88 lb).
- Participant has active infection.
- Participant has had a significant exacerbation of COPD or has required mechanical ventilation within 4 weeks of screening.
- The participant with clinically relevant uncontrolled medical condition not associated with COPD.
- Participant has documented history of uncontrolled heart failure.
- Participant has pulmonary hypertension due to left heart condition.
- Participant has atrial fibrillation or significant congenital heart defect/disease.
- Participant has initiated pulmonary rehabilitation within 3 months of screening.
- Participant is allergic to bovine or porcine products.
- Participant has evidence of active malignancy, or prior history of active malignancy that has not been in remission for at least 5 years.
- Participant has a life expectancy of < 6 months.
Sites / Locations
- David Geffen School of Medicine at UCLA
- Los Angeles Biomedical Research Institute at Harbor UCLA Medical Center
- American Health Research
- Upstate Pharmaceutical Research
- Spartanburg Medical Research
- Vermont Lung Center, University of Vermont
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Prochymal™
Placebo
Arm Description
Participants received Prochymal™ a total of 400×10^6 cells, intravenous (IV) infusions on Days 0, 30, 60, and 90.
Participants received placebo-matching IV infusions on Days 0, 30, 60 and 90.
Outcomes
Primary Outcome Measures
Number of Participants with Adverse Events (AEs)
Secondary Outcome Measures
Change from Baseline in Pulmonary Function Test: Forced Expiratory Volume (FEV)1 at Year 1 and Year 2
Change from Baseline in Pulmonary Function Test: Forced Expiratory Volume (FEV)1 %predicted at Year 1 and Year 2
Change from Baseline in Pulmonary Function Test: Forced Vital Capacity (FVC) at Year 1 and Year 2
Change from Baseline in Pulmonary Function Test: Forced Vital Capacity (FVC) %predicted at Year 1 and Year 2
Change from Baseline in Pulmonary Function Test: Forced Expiratory Volume to Forced Vital Capacity Ratio (FEV1/FVC) at Year 1 and Year 2
Change from Baseline in Diffusing capacity of the lung for carbon monoxide (DLCO) at Year 1 and Year 2
Change from Baseline in Diffusing capacity of the lung for carbon monoxide (DLCO) %predicted at Year 1 and Year 2
Change from Baseline in Alveolar Volume (VA) at Year 1 and Year 2
Change from Baseline in Diffusing capacity of the lung for carbon monoxide to Alveolar Volume ratio (DLCO/VA)at Year 1 and Year 2
Change from Baseline in Functional residual capacity (FRC) at Month 6
Change from Baseline in Total Lung Capacity (TLC) at Month 6
Change from Baseline in Residual Volume (RV) at Month 6
Change from Baseline in Airway Resistance (RAW) at Month 6
Change from Baseline in 6-Minute Walk Test at Year 1 and Year 2
Change from baseline in the total distance walked in 6 minutes was reported.
Change from Baseline in Borg Dyspnea Scale at Year 2
Change from Baseline in Health-related quality of life: St George's Respiratory Questionnaire (SGRQ) at Year 1 and Year 2
Change from Baseline in Physician Global Assessment Scale at Year 1 and Year 2
The physician evaluated the subject's global status as improved, unchanged, or worsened from pretreatment.
Time to COPD Exacerbation
Number of COPD Exacerbations
Change from Baseline in Pulmonary Hypertension at Month 6
Change from Baseline in Systemic Inflammation at Year 1 and Year 2
Changes in systemic inflammation was determined by C-Reactive Protein (CRP) assays.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00683722
Brief Title
PROCHYMAL™ (Human Adult Stem Cells) for the Treatment of Moderate to Severe Chronic Obstructive Pulmonary Disease (COPD)
Official Title
A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of PROCHYMAL™ (ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Intravenous Infusion for the Treatment of Subjects With Moderate to Severe Chronic Obstructive Pulmonary Disease (COPD)
Study Type
Interventional
2. Study Status
Record Verification Date
December 2021
Overall Recruitment Status
Completed
Study Start Date
March 20, 2008 (Actual)
Primary Completion Date
March 9, 2009 (Actual)
Study Completion Date
August 24, 2010 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mesoblast, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
The objective of the present study is to establish the safety and efficacy of multiple administrations of Prochymal™(ex-vivo cultured human adult mesenchymal stem cells) in participants with moderate to severe chronic obstructive pulmonary disease (COPD).
Detailed Description
COPD is currently the fourth leading cause of death in the United States. It is clear that there is a significant unmet medical need for safe and effective therapies to treat moderate to severe COPD. This patient population has a high mortality rate and requires frequent hospitalizations due to disease-related exacerbations. Based on severity distribution estimates, approximately 70% of all current COPD patient have either moderate or severe COPD. COPD has no known cure, thus current therapeutic intervention is aimed at providing relief of symptoms. Oxygen therapy is the only treatment that has been shown to improve survival. Smoking cessation has been shown to slow the rate of forced expiratory volume in 1 second (FEV1) decline and COPD progression. In general patient are treated with bronchodilators and inhaled corticosteroids, but again, these measures do not provide any significant benefit regarding disease progression or prognosis. The characteristics and biologic activity of Prochymal™, along with a good safety profile in human trials to date, suggest that Prochymal™ may be a good candidate for addressing this unmet medical need.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pulmonary Disease, Chronic Obstructive, Pulmonary Emphysema, Chronic Bronchitis
Keywords
COPD, Airflow Obstruction, Chronic, Chronic Airflow Obstruction, Chronic Obstructive Airway Disease, Chronic Obstructive Lung Disease, Chronic Obstructive Pulmonary Disease, Pulmonary Emphysema, Chronic Bronchitis, Mesenchymal Stem Cells (MSCs), Adult Human Stem Cells, Osiris, Prochymal™
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
62 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Prochymal™
Arm Type
Experimental
Arm Description
Participants received Prochymal™ a total of 400×10^6 cells, intravenous (IV) infusions on Days 0, 30, 60, and 90.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants received placebo-matching IV infusions on Days 0, 30, 60 and 90.
Intervention Type
Drug
Intervention Name(s)
Prochymal™
Intervention Description
IV infusion of ex- vivo cultured adult human mesenchymal stem cells.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
IV infusion of excipient of Prochymal™.
Primary Outcome Measure Information:
Title
Number of Participants with Adverse Events (AEs)
Time Frame
Up to 2 Years
Secondary Outcome Measure Information:
Title
Change from Baseline in Pulmonary Function Test: Forced Expiratory Volume (FEV)1 at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Pulmonary Function Test: Forced Expiratory Volume (FEV)1 %predicted at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Pulmonary Function Test: Forced Vital Capacity (FVC) at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Pulmonary Function Test: Forced Vital Capacity (FVC) %predicted at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Pulmonary Function Test: Forced Expiratory Volume to Forced Vital Capacity Ratio (FEV1/FVC) at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Diffusing capacity of the lung for carbon monoxide (DLCO) at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Diffusing capacity of the lung for carbon monoxide (DLCO) %predicted at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Alveolar Volume (VA) at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Diffusing capacity of the lung for carbon monoxide to Alveolar Volume ratio (DLCO/VA)at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Functional residual capacity (FRC) at Month 6
Time Frame
Baseline, Month 6
Title
Change from Baseline in Total Lung Capacity (TLC) at Month 6
Time Frame
Baseline, Month 6
Title
Change from Baseline in Residual Volume (RV) at Month 6
Time Frame
Baseline, Month 6
Title
Change from Baseline in Airway Resistance (RAW) at Month 6
Time Frame
Baseline, Month 6
Title
Change from Baseline in 6-Minute Walk Test at Year 1 and Year 2
Description
Change from baseline in the total distance walked in 6 minutes was reported.
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Borg Dyspnea Scale at Year 2
Time Frame
Baseline, Year 2
Title
Change from Baseline in Health-related quality of life: St George's Respiratory Questionnaire (SGRQ) at Year 1 and Year 2
Time Frame
Baseline, Year 1, Year 2
Title
Change from Baseline in Physician Global Assessment Scale at Year 1 and Year 2
Description
The physician evaluated the subject's global status as improved, unchanged, or worsened from pretreatment.
Time Frame
Baseline, Year 1, Year 2
Title
Time to COPD Exacerbation
Time Frame
Up to 2 Years
Title
Number of COPD Exacerbations
Time Frame
Up to 2 Years
Title
Change from Baseline in Pulmonary Hypertension at Month 6
Time Frame
Baseline, Month 6
Title
Change from Baseline in Systemic Inflammation at Year 1 and Year 2
Description
Changes in systemic inflammation was determined by C-Reactive Protein (CRP) assays.
Time Frame
Baseline, Year 1 and Year 2
10. Eligibility
Sex
All
Minimum Age & Unit of Time
40 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Participant must have a diagnosis of moderate or severe COPD.
Participant must have a post-bronchodilator FEV1/forced vital capacity (FVC) ratio < 0.7.
Participant must have a post-bronchodilator FEV1 % predicted value ≥ 30% and < 70%.
Participant must be between 40 and 80 years of age, of either sex, and of any race.
Participant must be a current or ex-smoker, with a cigarette smoking history of ≥ 10 years or > 10 pack-years.
Exclusion Criteria:
Participant has been diagnosed with asthma or other clinically relevant lung disease other than COPD (e.g. restrictive lung diseases, sarcoidosis, tuberculosis, idiopathic pulmonary fibrosis, bronchiectasis, or lung cancer).
Participant has been diagnosed with α1-antitrypsin deficiency.
Participant has a body mass greater than 150 kg (330 lb) or less than 40 kg (88 lb).
Participant has active infection.
Participant has had a significant exacerbation of COPD or has required mechanical ventilation within 4 weeks of screening.
The participant with clinically relevant uncontrolled medical condition not associated with COPD.
Participant has documented history of uncontrolled heart failure.
Participant has pulmonary hypertension due to left heart condition.
Participant has atrial fibrillation or significant congenital heart defect/disease.
Participant has initiated pulmonary rehabilitation within 3 months of screening.
Participant is allergic to bovine or porcine products.
Participant has evidence of active malignancy, or prior history of active malignancy that has not been in remission for at least 5 years.
Participant has a life expectancy of < 6 months.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mahboob Rahman, MD
Organizational Affiliation
Mesoblast, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
David Geffen School of Medicine at UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Los Angeles Biomedical Research Institute at Harbor UCLA Medical Center
City
Torrance
State/Province
California
ZIP/Postal Code
90502
Country
United States
Facility Name
American Health Research
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28207
Country
United States
Facility Name
Upstate Pharmaceutical Research
City
Greenville
State/Province
South Carolina
ZIP/Postal Code
29615
Country
United States
Facility Name
Spartanburg Medical Research
City
Spartanburg
State/Province
South Carolina
ZIP/Postal Code
29303
Country
United States
Facility Name
Vermont Lung Center, University of Vermont
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05446
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
33964910
Citation
Weiss DJ, Segal K, Casaburi R, Hayes J, Tashkin D. Effect of mesenchymal stromal cell infusions on lung function in COPD patients with high CRP levels. Respir Res. 2021 May 8;22(1):142. doi: 10.1186/s12931-021-01734-8.
Results Reference
derived
PubMed Identifier
23172272
Citation
Weiss DJ, Casaburi R, Flannery R, LeRoux-Williams M, Tashkin DP. A placebo-controlled, randomized trial of mesenchymal stem cells in COPD. Chest. 2013 Jun;143(6):1590-1598. doi: 10.1378/chest.12-2094.
Results Reference
derived
PubMed Identifier
22292600
Citation
Gross NJ. The COPD Pipeline XIV. COPD. 2012 Feb;9(1):81-3. doi: 10.3109/15412555.2012.646587. No abstract available.
Results Reference
derived
Learn more about this trial
PROCHYMAL™ (Human Adult Stem Cells) for the Treatment of Moderate to Severe Chronic Obstructive Pulmonary Disease (COPD)
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