A Phase 1 investigation will be performed to test the safety of two doses of bone marrow-derived MSCs (20,000,000 and 100,000,000) administered via peripheral intravenous infusion.
A Phase 1 investigation will be performed to test the safety of two doses of bone marrow-derived MSCs (20,000,000 and 100,000,000) administered via peripheralintravenous infusion. Group 1: 3 subjects will receive a single administration of allogeneic hMSCs: 20 million cells delivered via peripheral intravenous infusion Group 2: 3 subjects will receive a single administration of allogeneic hMSCs: 100 million cells delivered via peripheral intravenous infusion Interim safety analysis will be performed four weeks after the 1st subject is enrolled in each cohort. Continued safety and tolerability with review of adverse events (AEs) will be assessed at each visit. Efficacy parameters (pulmonary function tests, diffusing capacity (DLCO), lung volumes, 6-minute walk test (6MWT), and dyspnea/quality of life \[QOL\] questionnaires) will be assessed every 12 weeks until study completion. Clinical laboratory tests to assess safety will be performed at every visit.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
3
intravenous infusion of bone marrow-derived allogeneic stem cells
University of Miami Miller School of Medicine
Miami, Florida, United States
Number of Participant with treatment emergent serious adverse events
as defined as the incidence of any treatment-emergent serious adverse events; these are a composite of death, non-fatal pulmonary embolism, stroke, hospitalization for worsening dyspnea and clinically significant laboratory test abnormalities
Time frame: Week 4 post infusion
Difference in lung function
Difference in FEV1 Variability in morning peak expiratory flow measurements Difference in frequency of acute exacerbations defined as: hospitalizations, missed work days, and/or oral steroids for more than 3 days Decrease in fractional excretion of inhaled NO (FENO; less than 50 ppb)
Time frame: Participants will be followed from 1 week to an expected average of 48 weeks following infusion
Decrease in peripheral eosinophilia
Decrease in number of peripheral eosinophils
Time frame: Participants will be followed from 1 week to an expected average of 48 weeks following infusion
Difference in subject reported dyspnea and quality of life assessments
Difference in subject reported dyspnea and quality of life assessments: Asthma Control Test (ACT) and Asthma Control Questionnaire (ACQ)
Time frame: Participants will be followed from 1 week to an expected average of 48 weeks following infusion
Death from any cause
Death from any cause
Time frame: Participants will be followed from 1 week to an expected average of 48 weeks following infusion
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