Clinical trial NCT02161744: Safety, Tolerability and Preliminary Efficacy of Adipose Derive Stem Cells for Patients With COPD
Official title: A Phase I Open- Label Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Autologous Adipose Stem Cell (Adult Human) to Subjects With Chronic Obstructive Pulmonary Disease
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Chronic Obstructive Pulmonary Disease
Keywords: Adipose derived stem cells
Stromal vascular fraction cells
Pulmonary Function Tests
Chronic Obstructive Pulmonary Disease
Lead sponsor: Arkansas Heart Hospital
Sponsor class: OTHER
First posted: 2014-06-12
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Phase I open label study to assess safety and tolerability
Primary purpose: TREATMENT

Brief summary: The purpose of this study is: 1.- to assess the safety and tolerability of autologous adipose derived stem cells (aADSC) administered intravenously in patients with chronic obstructive pulmonary disease, and 2.- to assess if this therapy results in less decrease of lung function parameters (FEV1, FEV1/FVC and 6 min walking distance) compared with a control baseline of 6 weeks.

Patients will be followed up for 12 months after the therapy.

Detailed description: STUDY DESIGN This is a Phase I open- label single-dose study in subjects with significant COPD. This is an expanded safety cohort. The investigators will enroll a minimum of 30 subjects. 6 week data of FEV1, FEV1/FVC, DLCO and 6 min walking distance values will be collected in all patients who are enrolled in the study (Baseline and 6 weeks after). Prior to the stem cell treatment, the patient will be assessed for 6 weeks by pulmonary function tests and 6 min walk tests. Then, patients will receive the infusion of stromal vascular fraction cells containing the aADSC (single intravenous dose). The standard therapy of COPD patients will not be interrupted during the duration of the study. Safety will be monitored on an ongoing basis, and an interim safety review will be conducted by the Investigator(s) and Sponsor after the first 20 patients have been enrolled and treated.

Eligibility criteria: Inclusion Criteria:

1. Males and Females ≥18 years.
2. Cognitive competitiveness.
3. Diagnosis of at least moderate COPD (postbronchodilator FEV1/FVC \< 70% (\<0.7), FEV1 \< 80% predicted).
4. Diffusing capacity impairment (DLCO) assessed by single breath test (corrected for alveolar volume, 'DLVA').
5. Life expectancy greater than 12 months.
6. Ability to understand and provide signed informed consent, or have a designated legal guardian or spouse legally able and willing to make such decisions on the subject's behalf.
7. Reasonable expectation that patient will receive standard post-treatment care and attend all scheduled safety follow-up visits.
8. No changes in active pulmonary medications for heart failure during the two weeks prior enrollment.

10. Written informed consent. 11. Non-smoker or Past smoker, with 20 pack-years or more history

Exclusion Criteria:

1. Current smokers.
2. Unstable coronary disease.
3. Patients with industrial exposure to silica and/or asbestos or giant bullae.
4. Uncontrolled seizure disorder.
5. Dementia.
6. Presence of immune deficiency or autoimmune disease.
7. Alpha- 1 antitrypsin deficiency.
8. Cor pulmonale or diastolic heart failure NYHA class III or IV
9. Type 1 diabetes mellitus
10. Complicated type 2 diabetes mellitus.
11. Active tuberculosis or severe lung damage due to tuberculosis (extensive cavitation).
12. History of cancer within the past 5 years.
13. Clinically-significant hematologic, hepatic, or renal impairment as determined by screening clinical laboratory tests.
14. Systemic corticosteroids, cytostatics, immunosuppressive drug therapy (cyclophosphamide, methotrexate, cyclosporine, azathioprine, etc.), and DNA depleting or cytotoxic drugs taken within four weeks prior to study treatment.
15. Porphyria.
16. Allergy to sodium citrate or any "caine" type of local anesthetic.
17. Abnormal findings could include: known HIV infection or other immunodeficiency state, chronic active viral infection (such as hepatitis B or C), acute systemic infections (defined as patients undergoing treatment with antibiotics), gastrointestinal tract bleeding, or any severe or acute concomitant illness or injury.

Primary outcomes:
measure: Safety of adipose derived stem cells (ADSC) in Patient with COPD
timeFrame: 12 months
Secondary outcomes:
measure: Efficacy of ADSC in improving Shortness of Breath (SOB)
timeFrame: 2, 6 and 12 months
measure: Efficacy of ADSC In Pulmonary Function Test (PFTs)
timeFrame: 2, 6, 12 months
measure: Efficacy of adipose derived stem cell in 6 MWT
timeFrame: 2, 6, 12 months
measure: Efficacy of adipose derived stem cells in patient's perceived exertion.
timeFrame: 2, 6, 12 months
measure: Efficacy in Quality of life using George's Respiratory Questionnaire
timeFrame: 2, 6, 12 months
measure: Efficacy in Quality of life using the Chronic Respiratory questionnaires
timeFrame: 2, 6, 12 months
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Heart Hospital
state: Arkansas
city: Little Rock
country: United States
facility: Arkansas Site Management Services LLC
state: Arkansas
Study officials:
affiliation: Arkansas Heart Hospital
name: Jason Holder, MD
role: PRINCIPAL_INVESTIGATOR
References: