A Phase 1 interventional study of hMSCs in Bronchiectasis, sponsored by Marilyn Glassberg. Completed at 1 site in United States. Open to participants aged 30 Years to 87 Years. Per ClinicalTrials.gov, last updated 2019-08-28.
Sponsored by Marilyn Glassberg · Phase 1, Interventional, and Treatment
To demonstrate the safety of bone marrow-derived allogeneic human Mesenchymal Stem Cells (hMSCs) in patients with bronchiectasis receiving standard of care therapy, and to explore treatment efficacy
A Phase 1 investigation will be performed to test the safety of two doses of bone-marrow derived hMSCs (20,000,000 and 100,000,000) administered via peripheral intravenous infusion.
Group 1: 3 subjects will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion Group 2: 3 subjects will receive a single administration of allogeneic hMSCs: 1 x108 (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, lung diffusion capacity, 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.
High Resolution Computed Tomography (HRCT) scan will be performed at the baseline visit (if not done within three months prior to enrollment) and then at week 24.
368 studies on the registry are indexed under Bronchiectasis; 104 are open to participants now.
This study's enrollment of 6 is below the median of 60 across 233 interventional studies indexed under Bronchiectasis.
Browse Bronchiectasis studies →Marilyn Glassberg is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
3 patients will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion
Biological: hMSCs
3 patients will receive a single administration of allogeneic hMSCs: 1 x108 (100 million) cells delivered via peripheral intravenous infusion
Biological: hMSCs
intravenous infusion of bone marrow-derived allogeneic stem cells
Also known as: allogeneic mesenchymal stem cell
Number of Participant with treatment emergent serious adverse events
incidence of any treatment-emergent serious adverse events defined as the 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 Colony Forming Units (CFUs) in semiquantitative culture of sputum
Difference in CFUs in semiquantitative culture of sputum
Time frame: Participants will be followed from 1 week to an expected average of 24 weeks following infusion.
rate of decline of lung function
difference in absolute decline of forced expiratory volume at one second (FEV1) percent predicted
Time frame: Participants will be followed from 12 weeks to an expected average of 24 weeks following infusion.
frequency of acute exacerbations
increased cough and sputum production, fever, new or worsened dyspnea in less than 30 days, new or worsened hypoxemia in the absence of other identifiable causes
Time frame: Participants will be followed from 12 weeks to an expected average of 48 weeks following infusion.
reported dyspnea and quality of life assessment
using quality of life tool questionnaire QOL-B version 2
Time frame: Participants will be followed from 4 weeks to an expected average of 48 weeks following infusion.
death from any cause
death from any cause
Time frame: Participants will be followed for the duration of the trial, which is an expected average of 48 weeks.
Plan to share: No
This study is completed, as verified in Aug 2019. You cannot join it, but the record below documents what was studied.
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Marilyn Glassberg